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Lectures by The 2017 Award Recipients of Warren Alpert Foundation Prize in Cancer Immunology, October 5, 2017, HMS, 77 Louis Paster, Boston

Reporter: Aviva Lev-Ari, PhD, RN

Article ID #242: LIVE: Lectures by The 2017 Award Recipients of Warren Alpert Foundation Prize in Cancer Immunology, October 5, 2017, HMS, 77 Louis Paster, Boston. Published on 9/8/2017

WordCloud Image Produced by Adam Tubman

Top, from left: James Allison and Lieping Chen. Bottom, from left: Gordon Freeman, Tasuku Honjo (NOT ATTENDED), Arlene Sharpe.

Aviva Lev-Ari, PhD, RN was in attendance and covered this event LIVE

The 2017 Warren Alpert Foundation Prize has been awarded to five scientists for transformative discoveries in the field of cancer immunology.

Collectively, their work has elucidated foundational mechanisms in cancer’s ability to evade immune recognition and, in doing so, has profoundly altered the understanding of disease development and treatment. Their discoveries have led to the development of effective immune therapies for several types of cancer.

The 2017 award recipients are:

  • James Allison, professor of immunology and chair of the Department of Immunology, The University of Texas MD Anderson Cancer Center – Immune checkpoint blockage in Cancer Therapy strictly Genomics based drug
  1. 2017 FDA approved a genomics based drug
  2. and co-stimulatory signals
  3. CTLA-4 blockade, CD28, AntiCTLA-4 induces regression of Transplantable Murine tumor
  4. enhance tumor-specific immune response
  5. Fully antibody human immune response in 10,000 patients – FDA approved 2011
  6. Metastatic melanoma – 3 years survival, programmed tumor death, PD-1, MHC-A1
  7. Ipi/Nivo vs. Ipi – combination – 60% survival vs Ipi alone
  8. Anti CTA4 vs Anti-PD-1
  9. responsive T cell population – MC38 TILs
  10. MC38 Infiltrating T cell populations: T-reg, CD4, Effector, CD8, NKT/gamma-delta
  11. Checkpoint blockage modulates infiltrating T cell population frequencies
  12. T reg correlated with Tumor growth
  13. Combination therapy lead to CURE survival at 80% rate vs CTAL-4 40% positive outcome

Not Attended — Tasuku Honjo, professor of immunology and genomic medicine, Kyoto University – Immune regulation of Cancer Therapy by PD-1 Blockade

 

  • Lieping Chen, United Technologies Corporation Professor in Cancer Research and Professor of immunobiology, of dermatology and of medicine, Yale University – Adoptive Resistance: Molecular Pathway t Cancer Therapy – focus on solid tumors
  1. Enhancement – Enhance normal immune system – Co-stimulation/Co-inhibition Treg, and Cytokines, adoptive cell therapy, Lymphoid organs stores
  2. Normalization – to correct defective immune system – normalizing tumor immunity, diverse tumor escape mechanisms
  3. Anti-PD therapy: regression of large solid tumors: normalizing tumor immunity targeting tumor microenvironment: Heterogeneity, functional modulation, cellular and molecular components – classification by LACK of inflamation, adaptive resistance, other inhibitory pathways, intrinsic induction
  4. avoid autoimmune toxicity,
  5. Resetting immune response (melanoma)
  6. Understad Resistance: Target missing resistance or Adaptive resistance Type II= acquired immunity
  • Gordon Freeman, professor of medicine, Dana-Farber Cancer Institute, Harvard Medical School – PD-L1/PD-1 Cancer Immunotherapy
  1. B7 antibody
  2. block pathway – checkpoint blockage, Expand the T cells after recognition of the disease. T cell receptor signal, activation, co -stimulatory: B71 molecule, B72 – survival signals and cytokine production,.Increased T cell proliferation,
  3. PDL-1 is a ligand of PD 1. How T cell die? genes – PD1 Gene was highly expressed,
  4. Interferon gamma upregulate PD-L1 expression
  5. Feedback loop Tumor – stimulating immune response, interferon turn off PD1
  6. PD-L1 and PD-L2 Expression: Interferom
  7. Trancefuctor MHC, B7-2
  8. PD-L! sisgnat inhibit T-cell activation: turn off Proliferation and cytokine production — Decreasing the immune response
  9. T cell DNA Content: No S-phase devided cell
  10. PD-L1 engagement of PD-1 results in activation : Pd-1 Pathway inhibits T Cell Actiivation – lyposite motility,
  11. Pd-L2 is a second ligand for PD-1 and inhibits T cell activation
  12. PDl-1 expression: BR CA, Ovarian, Colonol-rectal, tymus, endothelial
  13. Blockage of the Pathway – Immune response enhanced
  14. Dendritic cells express PD-L1, PD-L2 and combination of Two, Combination was best of all by increase of cytokine production, increasing the immune response.
  15. PD-L1 blockade enhanced the immune response , increase killing and increased production of cytokines,
  16. anti-tumor efficacy of anti-PD-1/Pd-L1
  17. Pancreatic and colono-rector — PD-L, PDL1, PDL2 — does not owrkd.
  18. In menaloma: PD-1 works better than CYLA-4
  19. Comparison of Targeted Therapy: BRAF TKI vs Chemo high % but short term
  20. Immunotherapy – applies several mechanism: pre-existing anti-therapy
  21. Immune desert: PD=L does not work for them
  22. COMBINATION THERAPY: BLOCK TUMOR INVASION THEN STIMULATE IMMUNE RESPONSE — IT WILL WORK
  23. PD blockage + nutrients and probiotic
  24. Tumor Genome Therapy
  25. Tumore Immuno-evasion Score
  26. Antigens for immune response – choose the ones
  27. 20PD-1 or PD-L1 drugs in development
  28. WHO WILL THE DRUG WORK FOR?

 

  • Arlene Sharpe, the George Fabyan Professor of Comparative Pathology, Harvard Medical School; senior scientist, department of pathology, Brigham and Women’s Hospital – Multi-faceted Functionsof the PD-1 Pathway
  1. function of the pathway: control T cell activation and function of maintain immune tolerance
  2. protect tissues from damage by immune response
  3. T cell dysfunction during cancer anf viral infection
  4. protection from autoimmunity, inflammation,
  5. Mechanism by which PD-1 pathway inhibits anti-tumor immunity
  6. regulation of memoryT cell responce of PD-1
  7. PD-1 signaling inhibit anti-tumor immunity
  8. Compare: Mice lacking CD8-Cre- (0/5) cleared vs PD-1-/-5/5 – PD-1 DELETION: PARTIAL AND TIMED: DELETION OF PD-1 ON HALF OG TILS STARTING AT DAY 7 POSTTUMOR IMPLANTATION OF BOTH PD-1 AND PD-1 TILS: – Tamoxifen days 7-11
  9. Transcription profile: analysis of CD8+ TILs reveal altered metabolism: Fatty Acid Metabolism vs Oxidative Phosphorylation
  10. DOes metabolic shift: WIld type mouth vs PD-1-/_ P14: analyze Tumor cell killingPD-1-/- enhanced FAO increases CD8+ T cell tocicity
  11. Summary: T cell memory development and PD-1: T effectors vs T cell memory: Primary vs Secondary infection: In the absent of PD-1, CD8+ T cels show increase expansion of T cells
  12. INFLUENZA INFECTION: PRIMARY more virus in lung in PD-1 is lacking
  13. Acute infection: PD-1 controls memory T cell differentiation vs PD-1 increase expansion during effector phase BUT impaired persistence during memory phase: impaired cytokine production post re-challenge
  14. PD-1 immunotherapy work for patients with tumor: Recall Response and Primary response
  15. TIL density Primary vs Long term survivor – 5 days post tumor implantation – rechallenged long term survival
  16. Hot tumor vs Cold tumor – Deletion of PD-1 impairs T memory cell development

Opening Remarks: George Q. Daley, MD, PhD, DEAN, HMS

  • Scientific collaboration check point – avoid the body attacking itself, sabotaging the immune system
  • 1987 – Vaccine for HepB
  • Eight of the awardees got the Nobel Prize

 

Moderated by Joan Brugge, PhD, HMS, Prof. of Cell Biology

  • Evolution of concepts of Immunotherapy: William Coley’s Toxin streptoccocus skin infection.
  • 20th century: Immuno-surveilence, Immune response – field was dead in 1978 replaced by Immunotherapy
  • Rosenberg at NIH, high dose of costimulatory molecule prevented tumor reappearanceantbody induce tumor immunity–>> immune theraphy by check point receptor blockade – incidence of tumor in immune compromised mice – transfer T cell
  • T cell defficient, not completely defficient, self recognition of tumor,
  • suppress immmune – immune evasion
  • Michael Atkins, MD, Detupy Director, Georgetown-Lombardi, Comprehensive Cancer Center Clinical applications of Checkpoint inhibitors: Progress and Promise
  1. Overwhelm the Immune system, hide, subvert, Shield, defend-deactivating tumor trgeting T cells that ATTACK the immune system
  2. Immune system to TREAT the cancer
  3. Monotherapy – anti PD1/PD-L1: Antagonist activity
  4. Evading immune response: prostate, colcn
  5. MMR deficiency
  6. Nivolumab in relaped/Refractory HODGKIN LYMPHOMAS – over expression of PD-L1 and PDL2in Lymphomas
  7. 18 month survival better with Duv in Lung cancer stage 3 – anti PD-1- adjuvant therapy with broad effectiveness
  8. Biomarkers for pD-L1 Blockage
  9. ORR higher in PD-L1
  10. Improve Biomarkers: Clonality of T cells in Tumors
  11. T-effector Myeloid Inflammation Low – vs Hogh:
  12. Biomarker Model: Neoantigen burden vs Gene expression vs CD8+
  13. Tissue DIagnostic Labs: Tumor microenveironmenr
  14. Microbiome
  15. Combination: Nivo vs Nivo+Ipi is superior: DETERMINE WHEN TO STOP TREATMENT
  16. 15/16 stopped treatment – Treatment FREE SURVIVAL
  17. Sequencing with Standard Therapies
  18. Brain metastasis – Immune Oncology Therapy – crosses the BBB
  19. Less Toxic regimen, better toxicity management,
  20. Use Immuno therapy TFS
  21. combination – survival must be justified
  22. Goal: to make Cancer a curable disease vs cancer becoming a CHronic disease

Closing Remarks: George Q. Daley, MD, PhD, DEAN, HMS

The honorees will share a $500,000 prize and will be recognized at a day-long symposium on Oct. 5 at Harvard Medical School.

The Warren Alpert Foundation, in association with Harvard Medical School, honors trailblazing scientists whose work has led to the understanding, prevention, treatment or cure of human disease. The award recognizes seminal discoveries that hold the promise to change our understanding of disease or our ability to treat it.

“The discoveries honored by the Warren Alpert Foundation over the years are remarkable in their scope and potential,” said George Q. Daley, dean of Harvard Medical School. “The work of this year’s recipients is nothing short of breathtaking in its profound impact on medicine. These discoveries have reshaped our understanding of the body’s response to cancer and propelled our ability to treat several forms of this recalcitrant disease.”

The Warren Alpert Foundation Prize is given internationally. To date, the foundation has awarded nearly $4 million to 59 scientists. Since the award’s inception, eight honorees have also received a Nobel Prize.

“We commend these five scientists. Allison, Chen, Freeman, Honjoand Sharpe are indisputable standouts in the field of cancer immunology,” said Bevin Kaplan, director of the Warren Alpert Foundation. “Collectively, they are helping to turn the tide in the global fight against cancer. We couldn’t honor more worthy recipients for the Warren Alpert Foundation Prize.”

The 2017 award: Unraveling the mysterious interplay between cancer and immunity

Understanding how tumor cells sabotage the body’s immune defenses stems from the collective work of many scientists over many years and across multiple institutions.

Each of the five honorees identified key pieces of the puzzle.

The notion that cancer and immunity are closely connected and that a person’s immune defenses can be turned against cancer is at least a century old. However, the definitive proof and demonstration of the steps in this process were outlined through findings made by the five 2017 Warren Alpert prize recipients.

Under normal conditions, so-called checkpoint inhibitor molecules rein in the immune system to ensure that it does not attack the body’s own cells, tissues and organs. Building on each other’s work, the five award recipients demonstrated how this normal self-defense mechanism can be hijacked by tumors as a way to evade immune surveillance and dodge an attack. Subverting this mechanism allows cancer cells to survive and thrive.

A foundational discovery made in the 1980s elucidated the role of a molecule on the surface of T cells, the body’s elite assassins trained to seek, spot and destroy invaders.

A protein called CTLA-4 emerged as a key regulator of T cell behavior—one that signals to T cells the need to retreat from an attack. Experiments in mice lacking CTLA-4 and use of CTLA-4 antibodies demonstrated that absence of CTLA-4 or blocking its activity could lead to T cell activation and tumor destruction.

Subsequent work identified a different protein on the surface of T cells—PD-1—as another key regulator of T cell response. Mice lacking this protein developed an autoimmune disease as a result of aberrant T cell activity and over-inflammation.

Later on, scientists identified a molecule, B7-H1, subsequently renamed PD-L1, which binds to PD-1, clicking like a key in a lock. This was followed by the discovery of a second partner for PD-1—the molecule PD-L2—which also appeared to tame T-cell activity by binding to PD-1.

The identification of these molecules led to a set of studies showing that their presence on human and mouse tumors rendered the tumors resistant to immune eradication.

A series of experiments further elucidated just how tumors exploit the interaction between PD-1 and PD-L1 to survive. Specifically, some tumor cells appeared to express PD-L1, essentially “wrapping” themselves in it to avoid immune recognition and destruction.

Additional work demonstrated that using antibodies to block this interaction disarmed the tumors, rendering them vulnerable to immune destruction.

Collectively, the five scientists’ findings laid the foundation for antibody-based therapies that modulate the function of these molecules as a way to unleash the immune system against cancer cells.

Antibody therapy that targets CTLA-4 is currently approved by the FDA for the treatment of melanoma. PD-1/PD-L1 inhibitors have already shown efficacy in a broad range of cancers and have been approved by the FDA for the treatment of melanoma; kidney; lung; head and neck cancer; bladder cancer; some forms of colorectal cancer; Hodgkin lymphoma and Merkel cell carcinoma.

In their own words

“I am humbled to be included among the illustrious scientists who have been honored by the Warren Alpert Foundation for their contributions to the treatment and cure of human disease in its 30+ year history.  It is also recognition of the many investigators who have labored for decades to realize the promise of the immune system in treating cancer.”
        -James Allison


“The award is a great honor and a wonderful recognition of our work.”
         Lieping Chen



I am thrilled to have made a difference in the lives of cancer patients and to be recognized by fellow scientists for my part in the discovery of the PD-1/PD-L1 and PD-L2 pathway and its role in tumor immune evasion.  I am deeply honored to be a recipient of the Alpert Award and to be recognized for my part in the work that has led to effective cancer immunotherapy. The success of immunotherapy has unleashed the energies of a multitude of scientists to further advance this novel strategy.”
                                        -Gordon Freeman


I am extremely honored to receive the Warren Alpert Foundation Prize. I am very happy that our discovery of PD-1 in 1992 and subsequent 10-year basic research on PD-1 led to its clinical application as a novel cancer immunotherapy. I hope this development will encourage many scientists working in the basic biomedical field.”
-Tasuku Honjo


“I am truly honored to be a recipient of the Alpert Award. It is especially meaningful to be recognized by my colleagues for discoveries that helped define the biology of the CTLA-4 and PD-1 pathways. The clinical translation of our fundamental understanding of these pathways illustrates the value of basic science research, and I hope this inspires other scientists.”
-Arlene Sharpe

Previous winners

Last year’s award went to five scientists who were instrumental in the discovery and development of the CRISPR bacterial defense mechanism as a tool for gene editing. They were RodolpheBarrangou of North Carolina State University, Philippe Horvath of DuPont in Dangé-Saint-Romain, France, Jennifer Doudna of the University of California, Berkeley, Emmanuelle Charpentier of the Max Planck Institute for Infection Biology in Berlin and Umeå University in Sweden, and Virginijus Siksnys of the Institute of Biotechnology at Vilnius University in Lithuania.

Other past recipients include:

  • Tu Youyou of the China Academy of Chinese Medical Science, who went on to receive the 2015 Nobel Prize in Physiology or Medicine with two others, and Ruth and Victor Nussenzweig, of NYU Langone Medical Center, for their pioneering discoveries in chemistry and parasitology of malaria and the translation of their work into the development of drug therapies and an anti-malarial vaccine.
  • Oleh Hornykiewicz of the Medical University of Vienna and the University of Toronto; Roger Nicoll of the University of California, San Francisco; and Solomon Snyder of the Johns Hopkins University School of Medicine for research into neurotransmission and neurodegeneration.
  • David Botstein of Princeton University and Ronald Davis and David Hogness of Stanford University School of Medicine for contributions to the concepts and methods of creating a human genetic map.
  • Alain Carpentier of Hôpital Européen Georges-Pompidou in Paris and Robert Langer of MIT for innovations in bioengineering.
  • Harald zur Hausen and Lutz Gissmann of the German Cancer Research Center in Heidelberg for work on the human papillomavirus (HPV) and cancer of the cervix. Zur Hausenand others were honored with the Nobel Prize in Physiology or Medicine in 2008.

The Warren Alpert Foundation

Each year the Warren Alpert Foundation receives between 30 and 50 nominations from scientific leaders worldwide. Prize recipients are selected by the foundation’s scientific advisory board, which is composed of distinguished biomedical scientists and chaired by the dean of Harvard Medical School.

Warren Alpert (1920-2007), a native of Chelsea, Mass., established the prize in 1987 after reading about the development of a vaccine for hepatitis B. Alpert decided on the spot that he would like to reward such breakthroughs, so he picked up the phone and told the vaccine’s creator, Kenneth Murray of the University of Edinburgh, that he had won a prize. Alpert then set about creating the foundation.

To award subsequent prizes, Alpert asked Daniel Tosteson (1925-2009), then dean of Harvard Medical School, to convene a panel of experts to identify scientists from around the world whose research has had a direct impact on the treatment of disease.

SOURCE

https://hms.harvard.edu/news/warren-alpert-foundation-honors-pioneers-cancer-immunology

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Cleveland Clinic: Change at the Top,  Tomislav “Tom” Mihaljevic, M.D., as its next CEO and President to succeed Toby Cosgrove, M.D., effective Jan. 1, 2018

Reporter: Aviva Lev-Ari, PhD, RN

 

Cleveland Clinic’s Board of Governors and Board of Directors unanimously selected Dr. Mihaljevic based on the unanimous recommendation of a nomination committee chaired by Mr. Rich that conducted an extensive review of potential successors. Drs. Cosgrove and Mihaljevic will work on a transition process together through the end of the year. As of Jan. 1, 2018, Dr. Mihaljevic will assume the full duties of president and CEO, while Dr. Cosgrove will move to an advisory role to be determined by the Board of Directors and Dr. Mihaljevic.

A native of Croatia and a naturalized American citizen, Dr. Mihaljevic earned his medical degree from the University of Zagreb, before moving to the United States in 1995 to join Brigham and Women’s Hospital in Boston. He moved to Cleveland Clinic in 2004 as a cardiothoracic surgeon specializing in minimally invasive and robotically assisted cardiac surgeries – particularly robotic mitral valve repair – complex valve operations and reoperations, heart failure surgery, and heart transplantation. He helped to build Cleveland Clinic into the world’s largest robotic practice.

Dr. Mihaljevic has been on the editorial review board for prestigious medical journals. He is the author or co-author of more than 145 articles in medical and peer-reviewed scientific journals, and is the author of a numerous textbook chapters on robotic and minimally invasive mitral valve surgery, and heart valve disease. In 2005, Dr. Mihaljevic received a patent for a novel cardioscopy system for minimally invasive cardiac surgery. He earned the Cleveland Clinic Innovation Award in both 2006 and 2007.

Dr. Mihaljevic, 53, joined Cleveland Clinic in 2004 as a cardiothoracic surgeon specializing in minimally invasive and robotically assisted cardiac surgeries. Since 2015, Dr. Mihaljevic has served as CEO of Cleveland Clinic Abu Dhabi, overseeing the hospital’s strategy and operations as the first US multispecialty hospital to be replicated outside of North America, including directly managing the hospital’s Patient Experience and Strategy & Business Development programs.

Tomislav Mihaljevic, M.D. 

“Dr. Mihaljevic brings a depth of experience, first as an innovative, world-class surgeon and more recently as a hospital executive focused on healthcare quality and safety, patient experience and business strategy,” said Robert E. Rich Jr., chair of Cleveland Clinic’s Board of Directors. “By nearly every measure – quality, accessibility, finances, innovation, reputation – Cleveland Clinic has made unprecedented strides since Dr. Cosgrove became CEO and president in 2004. Following in his footsteps would be challenging for anybody, but Dr. Mihaljevic has the background, skills and vision to move Cleveland Clinic forward to even greater heights.”

SOURCE

https://newsroom.clevelandclinic.org/2017/09/01/tomislav-mihaljevic-m-d-named-cleveland-clinic-ceo-president/

 

Other related articles published in this Open Access Online Scientific Journal, include the following:

 

Articles on Minimally Invasive Surgery (MIS)

Cardiovascular Complications: Death from Reoperative Sternotomy after prior CABG, MVR, AVR, or Radiation; Complications of PCI; Sepsis from Cardiovascular Interventions

Author, Introduction and Summary: Justin D Pearlman, MD, PhD, FACC, and Article Curator: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2013/07/23/cardiovascular-complications-of-multiple-etiologies-repeat-sternotomy-post-cabg-or-avr-post-pci-pad-endoscopy-andor-resultant-of-systemic-sepsis/

 

Less is More: Minimalist Mitral Valve Repair: Expert Opinion of Prem S. Shekar, MD, Chief, Division of Cardiac Surgery, BWH – #7, 2017 Disruptive Dozen at #WMIF17

Reporter: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2017/05/17/less-is-more-minimalist-mitral-valve-repair-expert-opinion-of-prem-s-shekar-md-chief-division-of-cardiac-surgery-bwh-7-2017-disruptive-dozen-at-wmif17/

 

Left Main Coronary Artery Disease (LMCAD): Stents vs CABG – The less-invasive option is Equally Safe and Effective

Reporter: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2016/12/06/left-main-coronary-artery-disease-lmcad-stents-vs-cabg-the-less-invasive-option-is-equally-safe-and-effective/

 

New method for performing Aortic Valve Replacement: Transmural catheter procedure developed at NIH, Minimally-invasive tissue-crossing – Transcaval access, abdominal aorta and the inferior vena cava

Reporter: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2016/10/31/new-method-for-performing-aortic-valve-replacement-transmural-catheter-procedure-developed-at-nih-minimally-invasive-tissue-crossing-transcaval-access-abdominal-aorta-and-the-inferior-vena-cava/

 

Minimally Invasive Valve Therapy Programs: Recommendations by SCAI, AATS, ACC, STS

Reporter: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2014/05/19/minimally-invasive-valve-therapy-programs-recommendations-by-scai-aats-acc-sts/

 

Mitral Valve Repair: Who is a Patient Candidate for a Non-Ablative Fully Non-Invasive Procedure?

Author, and Content Consultant to e-SERIES A: Cardiovascular Diseases: Justin Pearlman, MD, PhD, FACC and Article Curator: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2013/11/04/mitral-valve-repair-who-is-a-candidate-for-a-non-ablative-fully-non-invasive-procedure/

 

Call for the abandonment of the Off-pump CABG surgery (OPCAB) in the On-pump / Off-pump Debate, +100 Research Studies

Curator: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2013/07/31/call-for-the-abandonment-of-the-off-pump-cabg-surgery-opcab-in-the-on-pump-off-pump-debate-100-research-studies/

 

3D Cardiovascular Theater – Hybrid Cath Lab/OR Suite, Hybrid Surgery, Complications Post PCI and Repeat Sternotomy

Curator: Aviva Lev-Ari, PhD, RN

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Emerging STAR in Molecular Biology, Synthetic Virology and Genomics: Clodagh C. O’Shea: ChromEMT – Visualizing 3D chromatin structure

Article ID #241: Emerging STAR in Molecular and Cell Biology, Synthetic Virology and Genomics: Clodagh C. O’Shea: ChromEMT – Visualizing 3D chromatin structure. Published on 8/31/2017

WordCloud Image Produced by Adam Tubman

Curator: Aviva Lev-Ari, PhD, RN

On 8/28/2017, I attend and covered in REAL TIME the CHI’s 5th Immune Oncology Summit – Oncolytic Virus Immunotherapy, August 28-29, 2017 Sheraton Boston Hotel | Boston, MA

I covered in REAL TIME this event and Clodagh C. O’Shea talk at the conference.

On that evening, I e-mailed my team that

“I believe that Clodagh C. O’Shea will get the Nobel Prizebefore CRISPR

11:00 Synthetic Virology: Modular Assembly of Designer Viruses for Cancer Therapy

Clodagh_OShea

Clodagh O’Shea, Ph.D., Howard Hughes Medical Institute Faculty Scholar; Associate Professor, William Scandling Developmental Chair, Molecular and Cell Biology Laboratory, Salk Institute for Biological Studies

Design is the ultimate test of understanding. For oncolytic therapies to achieve their potential, we need a deep mechanistic understanding of virus and tumor biology together with the ability to confer new properties.

To achieve this, we have developed

  • combinatorial modular genome assembly (ADsembly) platforms,
  • orthogonal capsid functionalization technologies (RapAd) and
  • replication assays that have enabled the rational design, directed evolution, systematic assembly and screening of powerful new vectors and oncolytic viruses.

Clodagh O’Shea’s Talk In Real Time:

  • Future Cancer therapies to be sophisticated as Cancer is
  • Targer suppresor pathways (Rb/p53)
  • OV are safe their efficacy ishas been limited
  • MOA: Specify Oncolytic Viral Replication in Tumor cells Attenuate – lack of potency
  • SOLUTIONS: Assembly: Assmble personalized V Tx fro libraries of functional parts
  • Adenovirus – natural & clinical advantages
  • Strategy: Technology for Assmbling Novel Adenovirus Genomes using Modular Genomic Parts
  • E1 module: Inactives Rb & p53
  • core module:
  • E3 Module Immune Evasion Tissue targeting
  • E4 Module Activates E2F (transcription factor TDP1/2), PI3K
  • Adenovirus promoters for Cellular viral replication — Tumor Selective Replication: Novel Viruses Selective Replicate in RB/p16
  • Engineering Viruses to overcome tumor heterogeneity
  • Target multiple & Specific Tumor Cel Receptors – RapAd Technology allows Re-targeting anti Rapamycin – induced targeting of adenovirus
  • Virus Genome: FKBP-fusion FRB-Fiber
  • Engineer Adenovirus Caspids that prevent Liver uptake and Sequestration – Natural Ad5 Therapies 
  • Solution: AdSyn335 Lead candidat AdSyn335 Viruses targeting multiple cells
  • Engineering Mutations that enhanced potency
  • Novel Vector: Homes and targets
  • Genetically engineered PDX1 – for Pancreatic Cancer Stroma: Early and Late Stage
On Twitter:
 

Engineer Adenovirus Caspids prevent Liver uptake and Sequestration – Natural Ad5 Therapies C. O’Shea, HHDI

 

Scientist’s Profile: Clodagh C. O’Shea

http://www.salk.edu/scientist/clodagh-oshea/

 

EDUCATION

BS, Biochemistry and Microbiology, University College Cork, Ireland
PhD, Imperial College London/Imperial Cancer Research Fund, U.K.
Postdoctoral Fellow, UCSF Comprehensive Cancer Center, San Francisco, U.S.A

VIDEOS

http://www.salk.edu/scientist/clodagh-oshea/videos/

O’Shea Lab @Salk

http://oshea.salk.edu/

AWARDS & HONORS

  • 2016 Howard Hughes Medical Institute Faculty Scholar
  • 2014 W. M. Keck Medical Research Program Award
  • 2014 Rose Hills Fellow
  • 2011Science/NSF International Science & Visualization Challenge, People’s Choice
  • 2011 Anna Fuller Award for Cancer Research
  • 2010, 2011, 2012 Kavli Frontiers Fellow, National Academy of Sciences
  • 2009 Sontag Distinguished Scientist Award
  • 2009 American Cancer Society Research Scholar Award
  • 2008 ACGT Young Investigator Award for Cancer Gene Therapy
  • 2008 Arnold and Mabel Beckman Young Investigator Award
  • 2008 William Scandling Assistant Professor, Developmental Chair
  • 2007 Emerald Foundation Schola

READ 

Clodagh C. O’Shea: ChromEMT: Visualizing 3D chromatin structure and compaction in interphase and mitotic cells | Science

http://science.sciencemag.org/content/357/6349/eaag0025

and 

https://www.readbyqxmd.com/keyword/93030

 

Clodagh C. O’Shea

In Press

Jul 27, 2017 – Salk scientists solve longstanding biological mystery of DNA organization

Sep 22, 2016 – Clodagh O’Shea named HHMI Faculty Scholar for groundbreaking work in designing synthetic viruses to destroy cancer

Oct 05, 2015 – Clodagh O’Shea awarded $3 million to unlock the “black box” of the nucleus

Aug 27, 2015 – The DNA damage response goes viral: a way in for new cancer treatments

Apr 12, 2013 – Salk Institute promotes three top scientists

Oct 16, 2012 – Cold viruses point the way to new cancer therapies

Aug 25, 2010 – Use the common cold virus to target and disrupt cancer cells?

Oct 22, 2009 – Salk scientist receives The Sontag Foundation’s Distinguished Scientist Award

May 15, 2008 – Salk scientist wins 2008 Beckman Young Investigator Award

Mar 24, 2008 – Salk scientist wins 2007 Young Investigator’s Award in Gene Therapy for Cancer

Read Full Post »

Shaun Coughlin from UCSF Cardiovascular Research Center to cardio group for the Novartis Institute for Biomedical Research in Cambridge, MA

Reporter: Aviva Lev-Ari, PhD, RN

 

The dean of the UCSF med school, Talmadge King, had this to say in his sendoff today:

Coughlin’s “research discoveries revealed a mechanism by which proteases regulate cellular behaviors including a key mechanism that controls blood platelet activation and clot formation. This work led to a new medical therapy for preventing heart attacks and strokes and has been honored by the American Heart Association’s Basic Science Award in 2003 and its Research Achievement Award in 2014. Among his numerous other awards are the Bristol-Myers Squibb Cardiovascular Research Award and the Distinguished Career Award from the International Society on Thrombosis and Haemostasis.”

SOURCE

https://endpts.com/top-ucsf-scientist-shaun-coughlin-joins-migration-to-big-pharma-leaping-to-novartis

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@PharmaceuticalIntelligence.com –  A Case Study on the LEADER in Curation of Scientific Findings

Author: Aviva Lev-Ari, PhD, RN

 

Multi-facets of the LPBI Group Intellectual Property (IP) ASSETS

 

 

 

  • Editorial & Publication of Articles in e-Books by Leaders in Pharmaceutical Business Intelligence: Contributions of Larry H Bernstein, MD, FCAP

https://pharmaceuticalintelligence.com/2014/10/16/editorial-publication-of-articles-in-e-books-by-leaders-in-pharmaceutical-business-intelligence-contributions-of-larry-h-bernstein-md-fcap/

  • Editorial & Publication of Articles in e-Books by Leaders in Pharmaceutical Business Intelligence: Contributions of Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2014/10/16/editorial-publication-of-articles-in-e-books-by-leaders-in-pharmaceutical-business-intelligence-contributions-of-aviva-lev-ari-phd-rn/

 

Innovations in e-Scientific Publishing Methodology Development accomplished by LPBI Group:

A.  Methodology for Curation of Scientific Findings – implementations for

  • Multi-Auhtors Authoring Cloud-based Platform

 

  • Journal Statistics – Interdisciplinary Journal covers interfaces of six domains (Life sciences, Pharmaceuticals, Medicine, Healthcare Policy, Biotech Intelligence and Medical Devices)

Curations of Scientific Findings of peer reviewed articles in top three journals in each of the Six domain

Curations written on a multi-Authoring platform by MDs, MD/PhDs, PharmD and PhDs, all 15 years after graduation of the advanced degree program, and each has a publication list before joined my team – they write clinical and medical interpretations of the scientific frontier as evidenced in the Scientific Finding section of published articles in Cell, Nature, Science, NEJM, other top journals in these six domains.

  1. Volume: 1.3 Million eReaders, ~5,150 Scientific articles, +500 categories of Research defining the Journal Ontology, 9,500 tags, 7,300, scientific comment on the articles submitted and exchange recorded between the Scientific community and our Team members
  2. Top two articles >25,000 eReaders
  3. Clicks on two Top Authors: >551,000
  4. from NIH +3,700 hits
  5. 2250 Journal subscribers by e-mail
  6. +6,200 Biotech Executive following up on LinkedIn
  • BioMed e-Series of e-Books in Medicine – 16 Volumes in Five e-Series: Cardiovascular, Genomics, Cancer, Immunology, Patient-centered Medicine

https://www.amazon.com/s/ref=dp_byline_sr_ebooks_9?ie=UTF8&text=Aviva+Lev-Ari&search-alias=digital-text&field-author=Aviva+Lev-Ari&sort=relevancerank

  • Team expertise
  1. e-Scientific Publishing: The Competitive Advantage of a Powerhouse for Curation of Scientific Findings and Methodology Development for e-Scientific Publishing – LPBI Group, A Case in Point
  2. FIVE years of e-Scientific Publishing @pharmaceuticalintellicence.com, Top Articles by Author and by e-Views >1,000, 4/27/2012 to 4/27/2017
  3. Innovations in electronic Scientific Publishing (eSP): Case Studies in Marketing eContent, Curation Methodology, Categories of Research Functions, Interdisciplinary conceptual innovations by Cross Section of Categories, Exposure to Frontiers of Science by Real Time Press coverage of Scientific Conferences

B.  Methodology for REAL TIME Coverage of Scientific Conferences using Social Media and Real Time e-Proceedings Generation: Conferences in Biotech, Life Sciences and Medicine

  • In House Developed Methodology for Real Time Press Coverage of Biotech Top International conferences – selective  topics covered at conferences lead to NEW Curations in the Journal

https://pharmaceuticalintelligence.com/press-coverage/

 

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    Cancer Therapies: Metabolic, Genomics, Interventional, Immunotherapy and Nanotechnology in Therapy Delivery (Series C Book 2)

    May 13, 2017 | Kindle eBook

    by Larry H. Bernstein and Demet Sag
    Subscribers read for free.
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    Etiologies of Cardiovascular Diseases: Epigenetics, Genetics and Genomics

    Nov 28, 2015 | Kindle eBook

    by Justin D. Pearlman MD ME PhD MA FACC and Stephen J. Williams PhD
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    Perspectives on Nitric Oxide in Disease Mechanisms (Biomed e-Books Book 1)

    Jun 20, 2013 | Kindle eBook

    by Margaret Baker PhD and Tilda Barliya PhD
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    Genomics Orientations for Personalized Medicine (Frontiers in Genomics Research Book 1)

    Nov 22, 2015 | Kindle eBook

    by Sudipta Saha PhD and Marcus W Feldman PhD
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    Metabolic Genomics & Pharmaceutics (BioMedicine – Metabolomics, Immunology, Infectious Diseases Book 1)

    Jul 21, 2015 | Kindle eBook

    by Larry H. Bernstein MD FCAP and Prabodah Kandala PhD
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    Milestones in Physiology: Discoveries in Medicine, Genomics and Therapeutics (Series E: Patient-Centered Medicine Book 3)

    Dec 26, 2015 | Kindle eBook

    by Larry H. Bernstein MD FACP and Aviva Lev-Ari PhD RN
    Subscribers read for free.
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    Cancer Biology and Genomics for Disease Diagnosis (Series C: e-Books on Cancer & Oncology Book 1)

    Aug 10, 2015 | Kindle eBook

    by Larry H Bernstein MD FCAP and Prabodh Kumar Kandala PhD
    Subscribers read for free.
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    Regenerative and Translational Medicine: The Therapeutic Promise for Cardiovascular Diseases

    Dec 26, 2015 | Kindle eBook

    by Justin D. Pearlman MD ME PhD MA FACC and Ritu Saxena PhD
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    Cardiovascular Original Research: Cases in Methodology Design for Content Co-Curation: The Art of Scientific & Medical Curation

    Nov 29, 2015 | Kindle eBook

    by Larry H. Bernstein MD FCAP and Aviva Lev-Ari PhD RN
    Subscribers read for free.
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Ido Sagi – PhD Student @HUJI, 2017 Kaye Innovation Award winner for leading research that yielded the first successful isolation and maintenance of haploid embryonic stem cells in humans.

Reporter: Aviva Lev-Ari, PhD, RN

 

Ido Sagi – PhD Student, Silberman Institute of Life Sciences, HUJI, Israel

  • Ido Sagi’s research focuses on studying genetic and epigenetic phenomena in human pluripotent stem cells, and his work has been published in leading scientific journals, including NatureNature Genetics and Cell Stem Cell.
  • Ido Sagi received BSc summa cum laude in Life Sciences from the Hebrew University, and currently pursues a PhD at the laboratory of Prof. Nissim Benvenisty at the university’s Department of Genetics in the Alexander Silberman Institute of Life Sciences.

The Kaye Innovation Awards at the Hebrew University of Jerusalem have been awarded annually since 1994. Isaac Kaye of England, a prominent industrialist in the pharmaceutical industry, established the awards to encourage faculty, staff and students of the Hebrew University to develop innovative methods and inventions with good commercial potential, which will benefit the university and society.

Publications – Ido Sagi

Comparable frequencies of coding mutations and loss of imprinting in human pluripotent cells derived by nuclear transfer and defined factors.
Cell Stem Cell 2014 Nov 6;15(5):634-42. Epub 2014 Nov 6.
The New York Stem Cell Foundation Research Institute, New York, NY 10032, USA; Naomi Berrie Diabetes Center & Department of Pediatrics, College of Physicians and Surgeons, Columbia University, New York, NY 10032, USA. Electronic address:

November 2014

 



Stem cells: Aspiring to naivety.
Nature 2016 12 30;540(7632):211-212. Epub 2016 Nov 30.
The Azrieli Center for Stem Cells and Genetic Research, Department of Genetics, Silberman Institute of Life Sciences, The Hebrew University of Jerusalem, Jerusalem 91904, Israel.
November 2016

Download Full Paper

SOURCE

Other related articles on Genetic and Epigenetic phenomena in human pluripotent stem cells published by LPBI Group can be found in the following e-Books on Amazon.com

e-Books in Medicine

https://www.amazon.com/s/ref=dp_byline_sr_ebooks_9?ie=UTF8&text=Aviva+Lev-Ari&search-alias=digital-text&field-author=Aviva+Lev-Ari&sort=relevancerank

9 results for Kindle Store : “Aviva Lev-Ari”

  • Product Details

    Etiologies of Cardiovascular Diseases: Epigenetics, Genetics and Genomics

    Nov 28, 2015 | Kindle eBook

    by Justin D. Pearlman MD ME PhD MA FACC and Stephen J. Williams PhD
    Subscribers read for free.
    Auto-delivered wirelessly
    Sold by: Amazon Digital Services LLC
  • Product Details

    Cancer Therapies: Metabolic, Genomics, Interventional, Immunotherapy and Nanotechnology in Therapy Delivery (Series C Book 2)

    May 13, 2017 | Kindle eBook

    by Larry H. Bernstein and Demet Sag
    Subscribers read for free.
    Auto-delivered wirelessly
    Sold by: Amazon Digital Services LLC
  • Product Details

    Perspectives on Nitric Oxide in Disease Mechanisms (Biomed e-Books Book 1)

    Jun 20, 2013 | Kindle eBook

    by Margaret Baker PhD and Aviva Lev-Ari PhD RN
    Subscribers read for free.
    Auto-delivered wirelessly
    Sold by: Amazon Digital Services LLC
  • Product Details

    Cancer Biology and Genomics for Disease Diagnosis (Series C: e-Books on Cancer & Oncology Book 1)

    Aug 10, 2015 | Kindle eBook

    by Larry H Bernstein MD FCAP and Prabodh Kumar Kandala PhD
    Subscribers read for free.
    Auto-delivered wirelessly
    Sold by: Amazon Digital Services LLC
  • Product Details

    Genomics Orientations for Personalized Medicine (Frontiers in Genomics Research Book 1)

    Nov 22, 2015 | Kindle eBook

    by Sudipta Saha PhD and Ritu Saxena PhD
    Subscribers read for free.
    Auto-delivered wirelessly
    Sold by: Amazon Digital Services LLC
  • Product Details

    Metabolic Genomics & Pharmaceutics (BioMedicine – Metabolomics, Immunology, Infectious Diseases Book 1)

    Jul 21, 2015 | Kindle eBook

    by Larry H. Bernstein MD FCAP and Prabodah Kandala PhD
    Subscribers read for free.
    Auto-delivered wirelessly
    Sold by: Amazon Digital Services LLC
  • Product Details

    Milestones in Physiology: Discoveries in Medicine, Genomics and Therapeutics (Series E: Patient-Centered Medicine Book 3)

    Dec 26, 2015 | Kindle eBook

    by Larry H. Bernstein MD FACP and Aviva Lev-Ari PhD RN
    Subscribers read for free.
    Auto-delivered wirelessly
    Sold by: Amazon Digital Services LLC
  • Product Details

    Regenerative and Translational Medicine: The Therapeutic Promise for Cardiovascular Diseases

    Dec 26, 2015 | Kindle eBook

    by Justin D. Pearlman MD ME PhD MA FACC and Ritu Saxena PhD
    Subscribers read for free.
    Auto-delivered wirelessly
    Sold by: Amazon Digital Services LLC
  • Product Details

    Cardiovascular Original Research: Cases in Methodology Design for Content Co-Curation: The Art of Scientific & Medical Curation

    Nov 29, 2015 | Kindle eBook

    by Larry H. Bernstein MD FCAP and Aviva Lev-Ari PhD RN
    Subscribers read for free.
    Auto-delivered wirelessly
    Sold by: Amazon Digital Services LLC

 

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Three Genres in e-Scientific Publishing AND Three Scientists’ Dilemmas

Curator: Aviva Lev-Ari, PhD, RN

 

That’s what I tell students. The way to succeed is to get born at the right time and in the right place. If you can do that then you are bound to succeed. You have to be receptive and have some talent as well.

Professor Sydney Brenner, a professor of Genetic medicine at the University of Cambridge and Nobel Laureate in Physiology or Medicine in 2002

 

 

Cell/Nature/Science

[CNS]

 Subscription-based Access

Open Access

  1. Online journals, to which scientists pay an upfront free to cover editing costs, which then ensure the work is available free to access for anyone in perpetuity

 

Curation of Scientific Findings

i.e., Kindle Direct Publishing [KDP] – Royalty-based system

  1. Free content to e-Readers
  2. Expert, Authors, Writers -Volunteers
  3. Editor -Voluneers
Confirming or disproving past studies Confirming or disproving past studies
Decades-long pursuit of a risky “moonshot” Decades-long pursuit of a risky “moonshot”
Trendy topics with Editors Trendy topics with Editors

 

Genres in e-Scientific Publishing

(A) Cell/Nature/Science

 – June 27, 2017

Elizabeth Dzeng — Feb 24th, 2014

  • http://www.cell.com/
  • http://www.sciencemag.org/
  • https://www.nature.com/
  • In 1998, Elsevier rolled out its plan for the internet age, which would come to be called “The Big Deal”. It offered electronic access to bundles of hundreds of journals at a time: a university would pay a set fee each year – according to a report based on freedom of information requests, Cornell University’s 2009 tab was just short of $2m – and any student or professor could download any journal they wanted through Elsevier’s website. Universities signed up en masse. …. Elsevier owned 24% of the scientific journal market, while Maxwell’s old partners Springer, and his crosstown rivals Wiley-Blackwell, controlled about another 12% each. These three companies accounted for half the market. (An Elsevier representative familiar with the report told me that by their own estimate they publish only 16% of the scientific literature.)  – June 27, 2017.  Elsevier published 420,000 papers last year, after receiving 1.5m submissions  – June 28, 2017 [numbers correction to 6/27/2017.]

(B) Open Access Journals and the Phenomenon

  1. Biochemistry
  2. Biophysics and Structural Biology
  3. Cancer Biology
  4. Cell Biology
  5. Computational and Systems Biology
  6. Developmental Biology and Stem Cells
  7. Epidemiology and Global Health
  8. Genomics and Evolutionary Biology
  9. Microbiology and Infectious Disease
  10. Neuroscience

(C) Curation of Scientific Findings

Scientists’ Dilemmas

(1) Confirming or disproving past studies

(2) Decades-long pursuit of a risky “moonshot”

(3) Trendy Topics with Editors 

 

@ PharmaceuticalIntelligence.com –  A Case Study on the LEADER in Curation of Scientific Findings

https://pharmaceuticalintelligence.com/2017/06/29/pharmaceuticalintelligence-com-a-case-study-on-the-leader-in-curation-of-scientific-findings/

Product Details

Cardiovascular Original Research: Cases in Methodology Design for Content Co-Curation: The Art of Scientific & Medical Curation

Nov 29, 2015 | Kindle eBook

by Larry H. Bernstein MD FCAP and Aviva Lev-Ari PhD RN
Subscribers read for free.
Auto-delivered wirelessly
Sold by: Amazon Digital Services LLC

 

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NIH announced its sixth class of Medical Research Scholars Program (MRSP) – 42 Students, 48% Females and 8 Minority Students

Reporter: Aviva Lev-Ari, PhD, RN

 

The National Institutes of Health has selected 42 talented and diverse students, representing 35 U.S.-accredited universities, for the sixth class of its Medical Research Scholars Program (MRSP). The MRSP received a record number of applications during the 2017-2018 application cycle. The 42 selected participants consist of 39 medical, two dental, and one veterinary student; 48 percent are female and eight individuals are from underrepresented minority groups. There are five second year, 35 third, and two fourth year students in the class; six of the 42 have had previous NIH research experience. The accepted scholars begin their MRSP fellowship in July/August of this year.

“These 42 scholars represent some of this country’s most promising future biomedical researchers and academic leaders.”

Frederick P. Ognibene, M.D., Director, Office of Clinical Research Training and Medical Education, NIH Clinical Center

The MRSP is co-sponsored by the NIH and other partners via contributions to the Foundation for the NIH.

The 42 participants for the 2017-2018 NIH MRSP include:

  1. Mairead Baker, Loyola University of Chicago, Stritch School of Medicine
  2. Fatima Barragan, Michigan State University, College of Human Medicine, East Lansing
  3. Jennifer Bayly, Rutgers, Robert Wood Johnson Medical School, Piscataway, New Jersey
  4. Jacqueline Boyle, University of Illinois College of Medicine at Peoria
  5. Rebecca Breese, the University of North Carolina at Chapel Hill School of Medicine
  6. Sonny Caplash, the University of Connecticut School of Medicine, Farmington
  7. Katherine Chen, the University of California, Irvine, College of Medicine
  8. Sophie Claudel, Wake Forest University School of Medicine, Winston-Salem, North Carolina
  9. Shavonne Collins, Meharry Medical College, Nashville
  10. Shazia Dharssi, Johns Hopkins University School of Medicine, Baltimore
  11. Joshua Diamond, the University of Virginia School of Medicine, Charlottesville
  12. Youssef Elnabawi, Tufts University School of Medicine, Boston
  13. Joseph Featherall, Cleveland Clinic Lerner College of Medicine of Case Western Reserve University
  14. Kathleen Fenerty, Indiana University School of Medicine, Indianapolis
  15. Samuel Gold, State University of New York, Downstate Medical Center College of Medicine, Brooklyn
  16. Morgan Graves, Georgetown University School of Medicine, Washington, D.C.
  17. Jacob Groenendyk, Washington University School of Medicine, St. Louis
  18. Russ Guidry, Louisiana State University School of Medicine, New Orleans
  19. Graham Hale, Jefferson Medical College of Thomas Jefferson University, Philadelphia
  20. Christopher Hampton, the University of Connecticut School of Medicine, Farmington
  21. Belen Hernandez, Colorado State University College of Veterinary Medicine, Fort Collins
  22. Christopher Hogden, the University of Iowa College of Dentistry, Iowa City
  23. Tommy Hu, Pennsylvania State University College of Medicine, Hershey
  24. Eileen Hu-Wang, Northwestern University The Feinberg School of Medicine, Chicago
  25. Sahar Khan, Cleveland Clinic Lerner College of Medicine of Case Western Reserve University
  26. Alyssa Kosturakis, the University of Texas School of Medicine at San Antonio
  27. Jason Lau, the University of Massachusetts Medical School, Worcester
  28. Andrew Lum, Tufts University School of Dental Medicine, Boston
  29. Uchenna Okoro, the University of Michigan Medical School, Ann Arbor
  30. Kristen Pan, the University of Cincinnati College of Medicine
  31. Priya Patel, Jacobs School of Medicine and Biomedical Sciences at the University at Buffalo
  32. Grant Randall, the University of Missouri – Kansas City School of Medicine
  33. Corinne Rauck, the University of Cincinnati College of Medicine
  34. Kareem Rayn, State University of New York, Downstate Medical Center College of Medicine, Brooklyn
  35. Isabelle Sanchez, the University of Illinois College of Medicine at Chicago
  36. Aakash Sathappan, the University of California, San Diego, School of Medicine
  37. Clayton Smith, Georgetown University School of Medicine, Washington, D.C.
  38. Dattanand Sudarshana, Cleveland Clinic Lerner College of Medicine of Case Western Reserve University
  39. Enock Teefe, Chicago Medical School at Rosalind Franklin University of Medicine and Science
  40. Alison Treichel, Jacobs School of Medicine and Biomedical Sciences at the University at Buffalo
  41. Fernando Vazquez, Dartmouth Medical School, Hanover, New Hampshire
  42. Jeannette Yu, Duke University School of Medicine, Durham, North Carolina

SOURCE

https://www.nih.gov/news-events/news-releases/nih-announces-2017-2018-medical-research-scholars-program-class

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2017 Raising Star: Dr. Dean Y. Li, MD, PhD – VP, Head Translational Medicine, Merck

Editor-in-Chief http://pharmaceuticalintelligence.comAviva Lev-Ari, PhD, RN

 

On May 1-3, 2017 –  I covered for the Press in Real Time the following event. The e-Reader is encouraged to click on each of the three links below.

 

  • World Medical Innovation Forum – CARDIOVASCULAR • MAY 1-3, 2017, BOSTON, MA

(a) Real Time Highlights and Tweets: Day 1,2,3: World Medical Innovation Forum – CARDIOVASCULAR • MAY 1-3, 2017, BOSTON, MA

(b) e-Proceedings for Day 1,2,3: World Medical Innovation Forum – CARDIOVASCULAR • MAY 1-3, 2017, BOSTON, MA

(c)  Tweets by @pharma_BI and @AVIVA1950 at World Medical Innovation Forum – CARDIOVASCULAR • MAY 1-3, 2017, BOSTON, MA

From this event, I plan to write about the 

Disruptive Dozen: 12 Technologies that will reinvent Cardiovascular Care

Moderator: Calum MacRae, MD, PhD

  • Chief of Cardiovascular Medicine, Brigham and Women’s Hospital
  • Associate Professor of Medicine, Harvard Medical School

Anthony Rosenzweig, MD

  • Chief, Cardiology Division, Massachusetts General Hospital
  • Professor of Medicine, Harvard Medical School

12. Aging and Heart Disease: Can we reverse the process?

11.Nanotechnologies for Cardiac Diagnosis and Treatment

10. Breaking the Code: Diagnosis and Therapeutic Potential of RNA

9. Expanding the Pool of Organs for Transplant

8. Finding Cancer therapies without Cardiotoxicity

7. Less is more: Minimalist Mitral Valve Repair

6. Understanding Why exercise works for Just about every thing

5. Power Play: The Future of Implantable Cardiac Devices

4. Adopting the Orphan of Heart Disease

3. Targeting Inflammation in cardiovascular Disease

2. Harnessing Big Data and Deep Learning for Clinical Decision Support

  1. Quantitative Molecular Imaging for Cardiovascular Phynotypes

 

Here, I wish to present the PROFILE of my own selection for the 2017 Raising Star: Dr. Dean Y. Li, MD, PhD  VP, Head Translational Medicine, Merck. His comments were per my judgement, among the most insightful.

 

Dr. Dean Y. Li, MD, PhD  VP, Head Translational Medicine, Merck

His lab has developed mouse models for three human vascular diseases:

  • supravalvular stenosis,
  • hereditary hemorrhagic telangiectasia,
  • cerebral cavernous malformations

Co-Founder of Early stage Biotech start ups:

  • Hydra Bioscience,
  • Navigen
  • Recursion Pharmaceuticals

Education

  • University of Chicago, BSc
  • Washington University in St. Louis, MD/PhD
  • University of Utah, 1994 as a Postdoctoral Fellow in Cardiology

 

Dr. Dean Y. Li, MD, PhD

5/2017 – VP, Head Translational Medicine, Merck

Past

Dr. Dean Y. Li is the H.A., and Edna Benning Endowed Professor of Internal Medicine and Director of the University of Utah Molecular Medicine (U2M2) Program and the MD/PhD Program. He received his Bachelor’s degree from the University of Chicago and his MD/PhD from Washington University in St. Louis. He began his career at the University of Utah in 1994 as a Postdoctoral Fellow in Cardiology. He received his first faculty appointment in 1996 which launched his career at the University of Utah. As is Associate Vice President for Research and Chief Scientific Officer for University of Utah Health Sciences, and Vice Dean for Research for the School of Medicine. Dr. Li is responsible for oversight of the research enterprise in the health sciences, including development and realization of a strategic plan for research. As Chief Scientific Officer, he is responsible for coordinating and maximizing the University’s health system investment in translational, clinical, and health care research leading to new treatments and health system innovations for patients.

Dr. Li is the recipient of multiple honors including the Established Investigator Award by the American Heart Association and Clinical Scientist Award in Translation Medicine from the Burroughs Wellcome Foundation and recently in May of 2014, he is the recipient of the Mark Brothers Award from Indiana University. In 2009 he received a Clinical Faculty Teaching Award and in 2010 he was inducted to the American Association of Physicians.

Dr. Li is a distinguished scientist and is internationally recognized for his groundbreaking research in vascular development and disease. His lab has developed mouse models for three human vascular diseases: supravalvular stenosis, hereditary hemorrhagic telangiectasia, and cerebral cavernous malformations. Through studying these diseases, he has identified a blood vessel protein that reversed or prevented age-related macular degeneration and diabetic retinopathy in mouse models. He also identified a family of proteins that accelerated blood vessel growth in ischemic mice and restored blood vessel growth in diabetic mice. Those discoveries have opened the door to developing drug therapies to treat those diseases.

Dr. Li has served as a reviewer for the AHA and for several study sections of the NIH. He is a member of the American Society of Clinical Investigators. He is the author of multiple manuscripts published in peer reviewed journals including Science and Nature. He is a current member of the AHA and NAVBO, co-founder of Hydra Bioscience, Navigen and Recursion Pharmaceuticals. His broad experiences and expertise in bench science, clinical medicine and biotechnology, combined with his success as a leader and mentor enable him to have a major impact on the School of Medicine’s mission to advance science and translate discoveries into clinical applications and better health care for patients.

SOURCE

http://www.u2m2.utah.edu/director/

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Top 50 Women in CRISPR : Women in CRISPR, Legal Status of Inventions and Declaration of the Heroes in CRISPR

Curator: Aviva Lev-Ari, PhD, RN

2.1.5.6

2.1.5.6   Top 50 Women in CRISPR : Women in CRISPR, Legal Status of Inventions and Declaration of the Heroes in CRISPR, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 2: CRISPR for Gene Editing and DNA Repair

Part 1: Top 50 Women in CRISPR : Women in CRISPR 

See List, below

SOURCE

Part 2: UPDATED – Status “Interference — Initial memorandum” – CRISPR/Cas9 – The Biotech Patent Fight of the Century: UC, Berkeley and Broad Institute @MIT

Reporter: Aviva Lev-Ari, PhD, RN

SOURCE

https://pharmaceuticalintelligence.com/2016/01/06/status-interference-initial-memorandum-crisprcas9-the-biotech-patent-fight-of-the-century/

Part 3: The Heroes of CRISPR

in CELL, December, 2015

Eric S. Lander1,2,3,*

1, Broad Institute of MIT and Harvard, 415 Main Street, Cambridge, MA 02142, USA

2Department of Biology, Massachusetts Institute of Technology, Cambridge, MA 02139, USA

3Department of Systems Biology, Harvard Medical School, Boston, MA 02115, USA

*Correspondence: lander@broadinstitute.org

Three years ago, scientists reported that CRISPR technology can enable precise and efficient genome editing in living eukaryotic cells. Since then, the method has taken the scientific community by storm, with thousands of labs using it for applications from biomedicine to agriculture. Yet, the preceding 20-year journey—the discovery of a strange microbial repeat sequence; its recognition as an adaptive immune system; its biological characterization; and its repurposing for genome engineering—remains little known. This Perspective aims to fill in this backstory—the history of ideas and the stories of pioneers—and draw lessons about the remarkable ecosystem underlying scientific discovery.

SOURCE

http://dx.doi.org/10.1016/j.cell.2015.12.041

REFERENCES

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Top 50 Women in CRISPR : Women in CRISPR

SOURCE
A B C D E F G H I J
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Women in CRISPR/Cas9 genome editing research – List Version 3
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First Name
Last Name Organisation Location Country Position Website
Twitter Handle
Field of Research
Research Interest
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Divaki Bhaya Stanford Univeristy Stanford, CA USA Professor https://dpb.carnegiescience.edu/labs/bhaya-lab
Evolution and Ecology – microbial diversity – Plant Biology
Research in my lab is driven by an interest in understanding how photosynthetic microorganisms perceive and evolve in response to environmental stressors, such as light, nutrients and viral attack.We work both with model organisms and with cyanobacteria in naturally occurring communities. Recently,we have started to develop synthetic biology-inspired approaches to use in cyanobacteria.
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Jill Banfield University of California Berkeley Berkeley, CA USA Professor http://nanogeoscience.berkeley.edu Evolution and Ecology – microbial diversity
The study system for this project is an aquifer adjacent to the Colorado River in Rifle, Colorado, USA.Research addresses knowledge gaps related to the roles of subsurface microbial communities in biogeochemical cycling. Given the link between the carbon cycle and global climate change, a particular interest in this work is the impact of microorganisms on carbon compounds buried in the terrestrial subsurface, both through respiration and carbon fixation.
5
Denis Bauer
Commonwealth Scientific and Industrial Research Organisation (CSIRO)
Sydney Australia Head of laboratory http://people.csiro.au/B/D/Denis-Bauer.aspx @allPowerde Computational biology – Technology development
Dr. Denis Bauer is the team leader of the transformational bioinformatics team in CSIRO’s ehealth program. Her expertise is in high throughput genomic data analysis, computational genome engineering, as well as Spark/Hadoop and high-performance compute system.
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Pilar Blancafort Harry Perkins Institute for Medical Research Perth Australia Associate Professor Cancer biology – Technology Development
The Blancafort laboratory focuses on the development of novel approaches to target cancers that are currently refractory to treatment and associated to poor outcome, such as triple negative breast cancers and ovarian cancers. At present, there are no targeted approaches to combat these tumors with chemotherapy and radiation the only treatment options. The laboratory generates novel functionalised molecules able to specifically target these tumors with minimal toxicity to normal cells. Our emphasis is in advanced stage metastatic tumors, which quasi invariably develop resistance. Ultimately we wish to revert the behavior of metastatic cells by sensitizing these treatment resistant tumors to chemotherapy regimes.
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Alexa Burger University of Zurich Zurich Switzerland
Senior postdoctoral fellow
http://www.imls.uzh.ch/en/research/mosimann/labmembers.html @aburger2009 Zebrafish – Technology development
CRISPR application in Zebrafish (ribonucleic complex and increase mutation efficiency)
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Emmanuelle Charpentier Max Plank Institute Berlin Germany Professor http://www.mpiib-berlin.mpg.de/research/regulation_in_infection_biology Host-pathogens interaction
Our research relates to the field of Molecular Infection Biology. We are overall interested in understanding the molecular mechanisms governing physiology-, virulence- and infection-associated processes in Gram-positive bacterial pathogens. We use a combination of genetic, genomic, molecular, biochemical, physiological and cell infection approaches to study mechanisms of gene expression at the transcriptional and post-transcriptional level in horizontal gene transfer, adaptation to stress, physiology or virulence. In particular, we do research on CRISPR, the adaptive immune system that protects bacteria against invading genetic elements; the small regulatory RNAs that interfere with bacterial pathogenicity; protein quality-control that regulates bacterial adaptation, physiology and virulence; and the mechanisms of bacterial recognition by immune cells.
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Sylvia Comporesi Kings College London London UK Lecturer https://silviacamporesiresearch.org/about/ @silviacomporesi Bioethics
I am a bioethicist with an interdisciplinary background in medical biotechnologies, ethics and philosophy. I am a tenured Lecturer (the UK equivalent to Assistant Professor) in Bioethics & Society in the Department of Global Health & Social Medicine (formerly, Social Science, Health & Medicine) at King’s College London, where I direct the Master’s in Bioethics & Society.
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Elena Conti Max Plank Institute Martinsried Germany
Group leader and Director
http://www.biochem.mpg.de/4877968/Research Structural Biology – RNA biology
Our group has a long-standing interest in RNA metabolism, with a particular focus on the molecular mechanisms of eukaryotic RNA transport and degradation.
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Jennifer Doudna University of California Berkeley Berkeley, CA USA Professor http://rna.berkeley.edu/index.html @doudna_lab RNA biology – Adaptive immunity
Exploring molecular mechanisms of RNA-mediated gene regulation
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Caixia Gao Chinese Academy of Science Beijing China Professor http://enpcce.genetics.cas.cn/PN/CXG/ACXG/ Plant biology (Wheat) – Technology development
The main research goal of our laboratory is to develop high-throughput transgene technologies for common wheat (Triticumaestivum L.) and maize (Zea mays) and other major crops to satisfy the needs of crop improvement and gene discovery.
13
Carine Giovanangeli Museum National d’Histoire Naturelle Paris France Director of Research http://biophysique.mnhn.fr/site/Modifications+génomiques+et+réponses+cellulaires DNA repair mechanisms – Technology development
Nowadays, we are mainly focusing on novel artificial DNA binding domains, the TALE repeats (transcription-activator like effector) and CRISPR/Cas9 system. We use the CRISPR/Cas or TALE as nucleases (TALEN) to study DNA repair in mammalian cells as well as DNA probes to study genome dynamics (see Repeated DNA sequences and chromatin).
14
Natalia Gomez-Ospina Stanford Univeristy Stanford, CA USA Clinical Instructor https://med.stanford.edu/profiles/natalia-gomez-ospina?tab=bio Stem cell biology – Clinical therapy
Dr. Gomez-Ospina was born and raised in Medellin, Colombia. She began her undergraduate studies in petroleum engineering at the Universidad Nacional de Colombia before moving to Colorado. She double majored at the University of Colorado Boulder, completing her bachelor’s degree in Molecular Cellular and Developmental Biology as well as Biochemistry. She graduated summa cum laude and wrote an honors thesis entitled “Role of the quiescent center in the regeneration of the root cap in Zea Mays.” She then completed her combined MD, PhD at Stanford Medical School, where her PhD work focused on understanding the novel functions of voltage-gated calcium channels. Her PhD thesis, “The calcium channel CACNA1C gene: multiple proteins, diverse functions,” was published in Cell. After completion of her dual degrees, she did her preliminary year in internal medicine at Santa Barbara Cottage hospital before starting residency in Dermatology at Johns Hopkins Hospital. She completed residency in Medical Genetics at Stanford Hospital and clinics. She is currently doing her post-doctoral research with Dr. Matthew Porteus in Pediatric Stem Cell transplantation, where she is developing a genome editing strategy in stem cells as a curative therapy for metabolic diseases. In addition to her research, Dr. Gomez-Ospina is a clinical instructor in Medical Genetics. For her clinical practice she sees patients with suspected genetic disorders, and is also in charge of the enzyme replacement service for lysosomal storage disorders at Lucile Packard Children’s hospital. She has been the lead author in research studies in The New England Journal of Medicine, Cell, Nature Communications, and American Journal of Medical Genetics.
15
Asma Hatoum-Aslan The University of Alabama Tuscaloosa, AL USA Assistant Professor http://bsc.ua.edu/asma-hatoum-aslan/ @crisprcas10 Host-pathogens interaction
Bacterial infectious diseases are a major cause of mortality worldwide. The rise in antibiotic resistant infections, coupled with the sharp decline in the discovery of new and clinically useful classes of antibiotics, underscores an urgent need for alternative strategies to combat bacterial infections. Small noncoding RNA pathways have recently been recognized as important regulators of bacterial pathogenesis, and the challenge lies in gaining a detailed understanding of these processes. My research uses the tools of biochemistry and molecular genetics to unravel the mechanisms of small RNA-mediated pathways and enable the development of novel anti-microbial therapeutics.
16
Rachel Haurwitz Caribou Biosciences Berkeley, CA USA
President and Chief Executive officer
http://cariboubio.com/about-us/management-team Biotech – Technology development
Rachel is a co-founder of Caribou Biosciences and has been President and CEO since its inception. She has a research background in CRISPR-Cas biology, and is also a co-founder of Intellia Therapeutics. In 2014, she was named by Forbes Magazine to the “30 Under 30” list in Science and Healthcare, and in 2016, Fortune Magazine named her to the “40 Under 40” list of the most influential young people in business. Rachel is an inventor on several patents and patent applications covering multiple CRISPR-derived technologies, and she has co-authored scientific papers in high impact journals characterizing CRISPR-Cas systems. Rachel earned an A.B. in Biological Sciences from Harvard College, and received a Ph.D. in Molecular and Cell Biology from the University of California, Berkeley.
17
Sara Howden Murdoch Children Research Institute Melbourne Australia Senior Research Fellow Stem cell biology – Technology development
Around 10-20% of kidney disease is inherited. In children with kidney disease, this is closer to 50% although in many instances, the disease-causing mutation is unknown, therefore limiting treatment options. In our research group, we investigate the genes required for normal kidney development and what happens as a result of genetic or environmental damage during development. This knowledge is used to try to recreate kidney stem cells. We have developed methods for generating mini-kidneys from human stem cells that represent models of the human organ. We hope to use these mini-kidneys to screen drugs for kidney toxicity, as models with which to understand kidney disease, to generate cells for the treatment of kidney disease and eventually to bioengineer replacement organs.
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Nina Hoyland-kroghsbo Princeton University Princeton, NJ USA Postdoctoral fellow http://molbiolabs.princeton.edu/bassler/members Host-pathogens interaction
Research Interest: The global threat of multi-drug resistant bacteria urgently demands alternatives to conventional antibiotics. Two promising alternatives to traditional antibiotics are bacteriophage (phage) therapy and inhibitors of bacterial cell-cell communication, known as quorum sensing (QS). Bacteria in high cell density maximally engage in QS. These cells are particularly vulnerable to phage infections, which could rapidly spread and kill the population. QS-control of antiphage activities would enable bacteria to specifically activate defenses when they are at the highest risk of infection. I am investigating to what extent bacteria use QS to regulate their antiphage defenses. Whereas QS-inhibitory compounds are generally studied for their capacity to inhibit bacterial virulence, I will study whether they additionally have the ability to increase the vulnerability of pathogenic bacteria to phages.
19
Danwei Huangfu Memorial Sloan Kettering New York, NY USA Head of laboratory https://www.mskcc.org/research-areas/labs/danwei-huangfu Stem cell biology – Technology development
The ability to program naïve cells or to reprogram differentiated cells into particular fates will open the door to the discovery of novel therapeutics for diseases such as diabetes. The goal of my lab is to understand the fundamental principles that govern the identity of a cell, and to use these principles to manipulate cell fates for regenerative medicine. In pursuit of this goal, we employ a variety of approaches including cellular programming and reprogramming through gene transduction, directed differentiation of embryonic stem (ES) cells, chemical screening, mouse genetics, adult tissue injury and regeneration, and tissue/cell transplantation.
20
Maria Jasin Memorial Sloan Kettering New York, NY USA Head of laboratory https://www.mskcc.org/research-areas/labs/maria-jasin DNA repair mechanisms – DSB
Human chromosomes are constantly assaulted by challenges to their integrity as a result of either environmental agents that damage DNA or from normal DNA metabolism. The failure to repair damaged DNA faithfully is ultimately responsible for many human diseases, especially cancer. This laboratory focuses on the repair of 1 particular lesion in DNA, the double-strand break (DSB). DSBs arise from agents, such as ionizing radiation, and can also occur spontaneously during DNA replication. Our emphasis is on repair of DSBs by homologous recombination, with a particular interest in the role of homologous recombination in maintaining genetic stability. Understanding the repair of DSBs is not only important for basic science and health concerns, but also impacts on molecular genetic manipulations of mammalian genomes
21
Josephin Johnston The Hasting Centre Garrison, NY USA Director of Research http://www.thehastingscenter.org/team/johnston/ @bioethicsjosie Bioethics
Josephine Johnston is an expert on the ethical, legal, and policy implications of biomedical technologies, particularly as used in human reproduction, psychiatry, genetics, and neuroscience.
22
Helene Jousset-Sabroux The Walter and Eliza Hall Institute for Medical Research Melbourne Australia Head of laboratory http://www.wehi.edu.au/people/hélène-jousset-sabroux
High Throughput Screening – Technology Development
The screening laboratory offers a wide range of expertise gained from both industrial and academic backgrounds, resulting in a professional ability to develop high capacity cellular or biochemical assays. We offer liquid handling robotics, plate readers and computing programs to increase the scale and speed of assays, and leverage automation to quickly assess the activity of a large number of compounds.
23
tamsin Lannagan University of Adelaide Adelaide Australia
Senior postdoctoral fellow
Cancer biology – Technology Development
My role within the group is to develop and assess novel mouse models of colorectal cancer, using colonoscopy techniques that are very similar to patient surveillance in humans. In addition, I am developing an in vitro method of growing mouse and human stem cells from the colon with their associated connective tissue. This will allow us to further investigate these support cells in normal growth and cancer. Both systems will be directly therapeutically relevant, allowing us to assess preclinical targeting of molecular pathways relevant to colorectal cancer.
24
Hong Li Florida State University Tallahassee, FL USA Professor http://biophysics.fsu.edu/hongli/ Structural Biology – RNA biology
A diverse range of RNA:protein, RNA:RNA and protein:protein interactions occur at the level of transcription and translation as well as post-transcriptional modifications. RNA:protein interactions are particularly interesting not only because they play important functional roles in assembly and biological processes, but also because the rules of their interactions are still poorly understood owing to the scarce structural data. Unlike DNA molecules, RNA can fold into a range of structures for interacting with proteins and small molecules. We hope, by providing exceptionally detailed images of the molecular events along the assembly and functional pathways, to unveil the underlying basis for assembly and functions involving RNA and partner proteins.
25
Jennifer Listgarten Microsoft Research Cambridge, MA USA Senior Researcher http://www.jennifer.listgarten.com Computational biology – Technology development
My area of expertise is in machine learning and applied statistics for computational biology. I’m interested in both methods development as well as application of methods to enable new insight into basic biology and medicine.
26
Shirley Liu Dana Farber Cancer Institute – Harvard Cambridge, MA USA Head of laboratory http://liulab.dfci.harvard.edu Computational biology – Technology development
We are developing the computational methods for the design (SSC), analysis (MAGeCK), hit prioritization (NEST), and visualization (VISPR) of genome-wide CRISPR screens. We are also using this technology to identify key genes in breast and prostate tumor progression and drug resistance. We also develop CRISPR screen platforms to understand the functions of enhancers and long-noncoding RNAs, and identify synthetic lethal gene pairs in cancer that leads to optimized cancer precision medicine.
27
Anita Marchfelder Ulm University Ulm Germany Head of laboratory https://www.uni-ulm.de/en/nawi/nawi-molbot/research/anita-marchfelder/ Host-pathogens interaction
All prokaryotic cells have to fend off foreign genetic elements like for instance viruses. To do that they have developed several different defence strategies. The recently discovered new defence strategy is the so called prokaryotic immune system also called CRISPR/Cas (CRISPR: clustered regularly interspaced short palindromic repeats, Cas: CRISPR-associated). It is adaptive, since cells can become immune against new invaders and it is heritable, since the information about the invader is stored in the genome. The CRISPR/Cas system consists of clusters of repetitive chromosomal DNA in which short palindromic DNA repeats are separated by spacers, the latter being sequences derived from the invader. In addition, a set of proteins, the Cas proteins, is involved in this defence reaction. We are investigating the CRISPR/Cas system in the halophilic archaeon Haloferax volcanii. Haloferax encodes a type I-B CRISPR/Cas system with eight Cas proteins and three CRISPR RNAs.
28
Karen Maxwell University of Toronto Toronto Canada Assistant Professor http://individual.utoronto.ca/maxwell_lab/ @theMaxwellLab Host-pathogens interaction
The Maxwell lab studies the phages that infect and kill the human bacterial pathogens Escherichia coli, Pseudomonas aeruginosa and Staphylococcus aureus. Infections caused by these bacteria create a significant disease burden, and the increasing incidence of antibiotic resistant infections caused by these pathogens is one of our most serious health threats.
29
Barbara J Meyer University of California Berkeley Berkeley, CA USA Head of laboratory http://mcb.berkeley.edu/labs/meyer/ Nematode – Technology development
Targeted Genome-editing Across Highly Diverged Nematode Species. Thwarted by the lack of reverse genetic approaches to enable cross-species comparisons of gene function, we established robust strategies for targeted genome editing across nematode species diverged by 300 MYR. In our initial work, a collaboration with Sangamo BioSciences, we used engineered nucleases containing fusions between the DNA cleavage domain of the enzyme FokI and a custom-designed DNA binding domain: either zinc-finger motifs for zinc-finger nucleases or transcription activator-like effector domains for TALE nucleases (TALENs). In those experiments, we allowed the DNA double-strand breaks to be repaired imprecisely by non-homologous end joining (NHEJ) to create mutations in precise locations.
30
Shondra Miller Washington University St Louis, MO USA Director of Research http://geic.wustl.edu/technology/ Stem cell biology – Technology development
The Genome Engineering and IPSC Center (GEiC) was formed by the consolidation of two pre-existing cores, the Genome Engineering Center and the Induced Pluripotent Stem cell (iPSC) core, both established by the Department of Genetics in the past few years. These two Centers were established to facilitate functional genomic studies through the use of patient-derived iPSCs and the generation of modified cells and organisms using genome editing technologies.
31
Hiromi Miura Tokai University School of Medicine Kanagawa Japan Assistant Professor https://www.researchgate.net/profile/Hiromi_Miura Mouse – Technology development
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Kathy Niakan The Francis Crick Institute London UK Head of laboratory https://www.crick.ac.uk/research/a-z-researchers/researchers-k-o/kathy-niakan/ Stem cell biology – Technology development
The allocation of cells to a specific lineage is regulated by the activities of key signalling pathways and developmentally regulated transcription factors. The focus of our research is to understand the influence of signalling and transcription factors on differentiation during early human development.
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Kate O’Connor-Giles University Wisconson Madison Madison, WI USA Head of laboratory http://oconnorgiles.molbio.wisc.edu Drosophila -Technology development
We are also developing genetic technologies for identifying and gaining genetic control of neuronal subtypes to determine their characterize their roles in neural circuits. Working with the laboratories of Jill Wildonger and Melissa Harrison, we recently adapted the CRISPR/Cas9 system for use in Drosophila. CRISPR is a novel technique that is revolutionizing genome engineering. Developed from bacteria where the CRISPR/Cas9 system functions in acquired immunity, CRISPR technology enables highly efficient and specific editing of targeted genomic sequences – opening the door to routine genome engineering. The many applications of CRISPR technology include modifying the genomes of model organisms to probe gene function, conferring disease resistance to agricultural organisms, and correcting disease-causing mutations in humans. We are capitalizing on this advance to develop novel genome engineering approaches that overcome current technological limitations to understanding neural circuits. Visit our flyCRISPR and flyCRISPR Optimal Target Finder sites for more details on our genome engineering work.
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April Pawluk University of California Berkeley Berkeley, CA USA Postdoctoral fellow http://rna.berkeley.edu/people.html @AprilPawluk Host-pathogens interaction
Bacteria and their cognate viruses, known as bacteriophages, are in a constant battle for survival. Among many mechanisms that bacteria possess to defend against bacteriophage infection, one of the most widespread and sophisticated is the CRISPR-Cas system. Setting CRISPR-Cas apart from other defence systems is the fact that it is an adaptive immunity system: one that can acquire the ability to target newly encountered invaders in a sequence-specific manner. Although much has been uncovered about the targeting mechanisms of CRISPR-Cas systems, very little is known about how they select and capture genetic snapshots of bacteriophages for later use as guides for the “seek and destroy” machinery. I leverage biochemical and structural biology approaches to investigate the CRISPR-Cas adaptation process in detail.
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Jennifer Phillips University of Oregon Eugene, OR USA Research Fellow http://zfin.org/ZDB-PERS-040915-1 @ClutchScience Zebrafish – Technology development
Our laboratory studies the molecular genetic basis of human diseases, particularly Usher syndrome, the leading cause of combined deafness and blindess, and other diseases of the eye and ear.
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Wenning Qin Biogen inc Cambridge, MA USA Director of Research https://www.biogen.com @wenningqin Mouse – Technology development
Wenning has been focusing on and exploring into genetic engineering technologies in her entire professional career. Her association includes Monsanto Biosciences, Pharmacia Corporation, Pfizer Incorporated and the Jackson Laboratory. She currently directs the Genetically Engineered Models group of Biogen, leveraging into genetic engineering to advance drug discovery pipeline for Biogen. Over the years, she acquired extensive knowledge and experience in design and creation of genetically engineered models, using random transgenesis, conventional gene targeting as well as CRISPR/Cas9 technology.
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Rakhi Rajan The University of Oklahoma Norman, OK USA Assistant Professor http://www.ou.edu/cas/chemistry/directory/faculty/rakhi-rajan.html RNA biology – Adaptive immunity
Protein-nucleic acid interactions are key to fundamental life processes such as DNA replication, transcription, recombination, and protein synthesis. Deciphering the mechanism of protein-nucleic acid interactions is invaluable for understanding human disease pathways and infections. The primary focus of my lab is to characterize protein-DNA/RNA interactions structurally, biochemically, and biophysically. The immediate emphasis is the study of the recently discovered bacterial and archaeal immune system, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats). CRISPR is an RNA-based adaptive immune system that inactivates foreign DNA/RNA entering the cell, based on the sequence similarity of small RNAs, called CRISPR RNA (crRNA) to the invading genetic element. The process requires several proteins called CRISPR associated (Cas) proteins. The CRISPR/Cas9 system has revolutionized the genome editing field due to the ease with which targeted double-stranded DNA breaks can be achieved in cells, using a guide RNA and Cas9 protein. The long-term goals of my laboratory are to understand the role of CRISPR/Cas system in pathogenicity and virulence of bacteria, characterize the mechanism of adaptation of bacteria to phage infection, and to determine the signaling mechanisms of the CRISPR/Cas system. We incorporate molecular biology, biochemistry, X-ray crystallography, and additional biophysical tools to characterize these protein-nucleic acid interactions.
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Dipali Sashital Iowa State University Ames, IA USA Assistant Professor http://www.sashitallab.org @dsashital RNA biology – Adaptive immunity
RNA-protein (RNP) complexes are central to many fundamental processes of gene regulation and genome maintenance in all kingdoms of life. The RNA components of these molecular machines often carry out diverse functions, acting as guide, template, scaffold, or catalyst. Despite this versatility, RNAs require protein partners to function, and the interactions that form between these components often dictate the overall activity of the RNP complex. Our lab is interested in understanding the molecular mechanisms underlying the function of RNPs from diverse cellular pathways. To that end, we combine a broad range of biochemical, structural and cellular tools to study RNA and protein structure, interactions and function.
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Nikki Shariat Gettysburg College Gettysburg, PA USA Assistant Professor https://sites.google.com/site/nikkishariat/home-1 RNA biology – Adaptive immunity
The Shariat Lab research interests are in prokaryote small RNA regulation and function, specifically in Clustered Regularly Interspaced Short Palindromic Repeats (CRISPRs). These elements are present in nearly half of all sequenced bacterial genomes and comprise several unique short sequences, called spacers, which are interspaced by conserved direct repeats. Spacers are derived from exogenous nucleic acids, such as bacteriophage genomes and plasmids. The spacers are transcribed into CRISPR RNAs (crRNAs), which are subsequently targeted to complementary nucleic acids, resulting in degradation of the target. Due to acquisition of new spacers, CRISPRs provide a remarkably dynamic adaptive immune system in both bacteria and archaea.
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Bettina Schmid Deutsches Zentrum fur Neurodegenerative Erkankungen Helmotz Germany Head of laboratory https://www.dzne.de/en/sites/munich/research-groups/schmid.html Zebrafish – Technology development
Our group uses the advantages of the zebrafish, Danio rerio, as an in vivo model system to address some of the unresolved questions in Alzheimer’s disease, Parkinson’s disease, Frontotemporal Lobar Degeneration (FTLD), and Amyotrophic lateral Sclerosis (ALS).
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Kimberley Seed University of California Berkeley Berkeley, CA USA Assistant Professor http://www.kimseedlab.com/#introduction Host-pathogens interaction
The ability of V. cholerae to prevent phage predation is critical for its evolutionary fitness and epidemic potential. In turn, as obligate bacterial parasites, phages must co-evolve to overcome this resistance or they will face extinction. Our research is aimed at understanding the bacterial immunity and opposing phage immune evasion strategies at play in this dynamic co-evolutionary arms race. We use comparative genomics and complementary molecular approaches to identify and experimentally validate such strategies in disease associated phage and V. cholerae isolates.
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Kaylene Simpson Peter McCallum Cancer Centre Melbourne Australia Associate Professor
High Throughput Screening – Technology Development
The Victorian Centre for Functional Genomics (VCFG) at Peter Mac offers biomedical researchers Australia-wide the ability to perform novel discovery-based functional interrogation all genes in the genome, or selected boutique collections using multiple platforms including CRISPR/cas9, small interfering RNA (siRNA), micro RNA (miRNA) and long non-coding RNA (lncRNA) and short hairpin RNA (shRNA).
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Joyce Van Eyck Cornell Univeristy Ithaca, NY USA Assistant Professor http://bti.cornell.edu/explore-bti/directory/joyce-van-eck/#research-overview Plant Biology (Tomato) – Technology Development
The focus of research in the Van Eck laboratory is biotechnological approaches to the study of gene function and crop improvement. For our studies, we apply several genetic engineering strategies to two major food crops: potato and tomato. The development of biotechnological techniques has made it possible to design and introduce gene constructs into plant cells and recover plants that express the introduced genes. Genes of interest to us have the potential to strengthen a plant’s resistance to disease, improve fruit characteristics, and enhance nutritional quality.
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Stineke Van Houte University of Exeter Exeter UK Research Fellow http://www.exeter.ac.uk/esi/people/researchandtechnical/van_houte/ Host-pathogens interaction
I am a biologist with a broad interest in host-parasite interactions, from an evolutionary, ecological and molecular perspective. Currently I work as a Marie-Curie fellow in the lab of Professor Angus Buckling on the evolution of immunity against virus infections in Pseudomonas bacteria. My PhD research at the Laboratory of Virology, Wageningen University (the Netherlands) focused on manipulation of host insect behaviour by baculoviruses, insect-specific viruses that cause lethal disease in caterpillars.
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Leslie Vosshall The Rockfeller Univeristy New York, NY USA Head of laboratory http://vosshall.rockefeller.edu @pollyp1 Insect – Technology development
The overall goal of work in our laboratory is to understand how complex behaviors are modulated by external chemosensory cues and internal physiological states. The lab takes a multi-disciplinary approach spanning cell biology, genetics, neurobiology and behavior. Our early focus has been to study how the brain interprets olfactory signals in the environment that signal food, danger, or potential mating partners. We have been studying these problems in three model organisms: the fly, the mosquito and the human. The majority of the early work in the laboratory was carried out in the genetically tractable vinegar fly, Drosophila melanogaster, which displays a rich repertoire of chemosensory behaviors despite having a nervous system with only 100,000 neurons. In this animal, we have studied the functional neuroanatomy of the olfactory system, how this system perceives sex pheromones, and the structure and function of the insect odorant receptors.
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Kan Wang Iowa State University Agron,IA USA Professor http://www.agron.iastate.edu/personnel/userspage.aspx?id=266 Plant biology (Maize) – Technology development
As the rapid development in plant genomics research identifies more genes, their functional analysis relies on strategies such as complementation, overexpression, or gene silencing. Plant genetic transformation is a critical technology required in the application of these strategies.
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Rachel Whitaker University of Illinois at Urbana Champaign Urbana, IL USA Associate Professor http://www.life.illinois.edu/whitaker/ Evolution and Ecology – Adaptive immunity
My lab combines population genomics with laboratory-based genetic and genomic experimental techniques to study the evolutionary ecology of microbial populations. We take a comparative approach, examining interactions within and between species using wild strains from natural populations isolated across spatial and temporal scales. Currently we are working on two critical forces that define the evolutionary process in all organisms: host-virus co-evolution and recombinational gene flow. We have a particular interest in how the unique biology of organisms in the Archaeal domain is reflected in genome architecture and how the CRISPR-Cas immune system functions in microbial populations.
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Susan Woods University of Adelaide Adelaide Australia Senior Research Fellow https://researchers.adelaide.edu.au/profile/susan.woods#career Cancer biology – Technology Development
Susan’s current project focuses on colorectal cancer. This is the second most common cancer type in Australia, costing us over $1 billion dollars annually. There are minimal effective treatments for advanced disease. The lab has recently identified a new stem cell that gives rise to a layer of cells that support the intestinal lining. We are investigating whether similar support cells can promote the formation of colorectal cancer from cells lining the intestine, and if we can prevent it using a new therapeutic approach.
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Luhan Yang eGenesis Cambridge, MA USA Co-founder and CSO http://www.egenesisbio.com/founding-team.html Biotech – Technology development
Luhan is leading the effort to eradicate PERVs from the porcine genome and engineer human compatibility in porcine cells. She previously developed the highly programmable genome-engineering tool, CRISPR/Cas9, for use in mammalian cells, and pioneered the first isogenic human stem cell lines to model human diseases at the tissue level. She was named among the “30 Under 30” in Science and Healthcare by Forbes Magazine (2014) and was a laureate of the “Young Entrepreneur Initiative” competition (2014). Luhan holds B.S. degrees in Biology and Psychology from Peking University and a Ph.D. in Human Biology and Translational Medicine from Harvard Medical School.
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Yan Zhang University of Michigan Ann Arbor, MI USA Assistant Professor https://medicine.umich.edu/dept/biochem/yan-zhang-p RNA biology – Technology development
CRISPR-Cas is a RNA-guided, genetic interference pathway in prokaryotes that enables acquired immunity against invasive nucleic acids. Nowadays, CRISPRs also provide formidable tools for facile, programmable genome engineering in eukaryotes. Cas9 proteins are the “effector” endonucleases for CRISPR interference; and have recently begun to be also recognized as important players in other aspects of bacterial physiology (e.g. acquisition of new spacers into CRISPRs, endogenous gene regulation, and microbial pathogenesis, etc.).My laboratory is broadly interested in CRISPR biology and mechanism. We will use Neisseria species as our model system, and E. coli and human cells as additional platforms. We employ complementary biochemical, microbiological, genetic and genomic approaches. We are also interested in working with the broader scientific community to develop and apply novel CRISPR-based tools to tackle diverse biological questions.

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