Archive for the ‘CRISPR/Cas9 & Gene Editing’ Category

GENE EDITING: Promises and Challenges: HSPH and NBC News Digital, Friday, May 19, 2017  Live webcast: 12:30-1:30pm ET

Reporter: Aviva Lev-Ari, PhD, RN



GENE EDITING: Promises and Challenges 

Presented jointly with NBC News Digital

Friday, May 19, 2017

Live webcast: 12:30-1:30pm ET


In labs and in clinical trials, scientists are seeking ways to rewrite DNA, a building block of life. Tools such as zinc-finger nucleases (ZFNs), TAL effector nucleases (TALENs) and, more recently, CRISPR/Cas9 have the power to seek out and replace faulty DNA. The possibilities seem almost limitless: with the ability to edit DNA at will, researchers theoretically could wipe out malaria-causing mosquitos, make disease- and pest-proof crops without the need for pesticides, and cure genetic diseases, such as sickle cell anemia and cystic fibrosis. Cancer is another target, with human clinical trials using CRISPR already underway, while, in separate efforts, HIV has been reportedly eliminated in mice thanks to the tool.

But scientists and ethicists alike are worried about the speed at which the gene editing field is moving — and the implications of the results. In this panel, we will discuss the promises and challenges presented by gene editing for individual and public health. What scientific and ethical hurdles must be overcome before tools like CRISPR and others can move safely and more widely out of the lab and into fields, farms, and hospitals? 



George Annas, Distinguished Professor at Boston University and Director of the Center for Health Law, Ethics & Human Rights at Boston University School of Public Health


Flaminia Catteruccia, Associate Professor of Immunology and Infectious Diseases, Harvard T.H. Chan School of Public Health


George Church, Professor of Genetics, Harvard Medical School, and Co-Founder, Editas and eGenesis



David Freeman, Editorial Director, NBC News MACH

Additional panelists may be announced.

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Send our panelists questions in advance to

We’ll be conducting a live chat on The Forum’s Gene Editing web page.

Tweet us @ForumHSPH  #GeneEditing

Forum video will be posted on-demand after the event.


From: “The Forum at Harvard T.H. Chan School of Public Health” <> on behalf of “The Forum at Harvard T.H. Chan School of Public Health” <>

Reply-To: “The Forum at Harvard T.H. Chan School of Public Health” <>

Date: Monday, May 15, 2017 at 2:36 PM

To: Aviva Lev-Ari <>

Subject: Gene Editing: Promises and Challenges


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Doudna and Charpentierand their teams to receive wide-ranging patents in many countries:  European Patent Office (EPO) and UK Intellectual Property Office – broad patent for CRISPR-Cas9 gene-editing technology to the University of California and the University of Vienna

Reporter: Aviva Lev-Ari, PhD, RN


The EPO patent will cover the single-guide CRISPR-Cas9 technology in cells of all types. The technology was invented by Jennifer Doudna, a UC Berkeley professor of molecular and cell biology, Charpentier, now director of the Max Planck Institute for Infection Biology in Berlin, and their colleagues. Applications include treatment of various human diseases, as well as veterinary, agricultural and other biotech applications. The European patent would cover some 40 countries, including France, Germany, Italy, Spain, the Netherlands and Switzerland.

The EPO has stated its intent to grant a patent with claims that encompass all cells, despite objections from third parties, including the Broad Institute, a joint research institute of Harvard University and the Massachusetts Institute of Technology.

“We are excited that this patent will issue based on the foundational research we published with Emmanuelle Charpentier and the rest of our team. We look forward to the continued applications of gene-editing technology to solve problems in human health and agriculture,” said Doudna, who is a Howard Hughes Medical investigator at UC Berkeley.

The UC patent application to the EPO was substantially the same as the UC patent application filed in the United States. In the U.S., UC claims covering the use of single-guide CRISPR-Cas9 technology in any setting were found to be allowable by the U.S. Patent & Trademark Office, and were placed in an interference with patents owned by the Broad Institute that cover use of the technology in eukaryotic cells. An interference is a formal legal proceeding before the Patent Trial and Appeal Board (PTAB) to determine who was the first to invent.

“We disagree with the recent PTAB decision to terminate the interference between claims of the UC and the Broad Institute, and we are keeping all of our options open, including the possibility of an appeal,” Penhoet said. “We remain confident that when the inventorship question is finally answered, the Doudna and Charpentier teams will prevail.”



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

Gene Editing Consortium of Biotech Companies: CRISPR Therapeutics $CRSP, Intellia Therapeutics $NTLA, Caribou Biosciences, ERS Genomics, UC, Berkeley (Doudna’s IP) and University of Vienna (Charpentier’s IP), is appealing the decision ruled that there was no interference between the two sides, to the U.S. Court of Appeals for the Federal Circuit, targeting patents from The Broad Institute.


Keyword Search: CRISPR – 247 articles in

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Gene Editing Consortium of Biotech Companies: CRISPR Therapeutics $CRSP, Intellia Therapeutics $NTLA, Caribou Biosciences, ERS Genomics, UC, Berkeley (Doudna’s IP) and University of Vienna (Charpentier’s IP), is appealing the decision ruled that there was no interference between the two sides, to the U.S. Court of Appeals for the Federal Circuit, targeting patents from The Broad Institute.


Curator: Aviva Lev-Ari, PhD, RN


See Background:

UPDATED – Status “Interference — Initial memorandum” – CRISPR/Cas9 – The Biotech Patent Fight of the Century


Source: Intellia Therapeutics, Inc.
  • Appeal to the U.S. Court of Appeals for the Federal Circuit seeks review and reversal of the Patent Trial and Appeals Board’s (PTAB) decision to terminate CRISPR/Cas9 interference
  • In parallel, the companies and their licensors plan to pursue additional patents in the U.S. and worldwide covering the CRISPR/Cas9 technology and its use in cellular and non-cellular settings, including eukaryotic cells

BASEL, Switzerland;

CAMBRIDGE, Massachusetts;

BERKELEY, California;

DUBLIN, Ireland,

April 13, 2017

(GLOBE NEWSWIRE) — CRISPR Therapeutics (NASDAQ:CRSP), Intellia Therapeutics (NASDAQ:NTLA), Caribou Biosciences and ERS Genomics announced today that The Regents of the University of California, the University of Vienna, and Dr. Emmanuelle Charpentier (collectively “UC”), co-owners of foundational intellectual property relating to CRISPR/Cas9 genome engineering, have appealed to the U.S. Court of Appeals for the Federal Circuit (the “Federal Circuit”) the decision by the Patent Trial and Appeal Board (“PTAB”) to terminate the interference between certain CRISPR/Cas9 patent claims owned by UC and patents and patent applications owned by the Broad Institute, Harvard University and the Massachusetts Institute of Technology (collectively, “Broad”).

In the appeal, UC is seeking review and reversal of the PTAB’s February 15, 2017 decision, which terminated the interference without determining which inventors actually invented the use of the CRISPR/Cas9 genome editing technology in eukaryotic cells. In its decision, the PTAB concluded that, although the claims overlap, the respective scope of UC and Broad’s claim sets as presented did not define the same patentable invention and, accordingly, terminated the interference without deciding which party first invented the use of the CRISPR/Cas9 technology in eukaryotic cells. UC is asking the Federal Circuit to review and reverse the PTAB’s decision.

In parallel with the appeal, UC is pursuing applications in the U.S. and other jurisdictions worldwide to obtain patents claiming the CRISPR/Cas9 technology and its use in non-cellular and cellular settings, including eukaryotic cells. Corresponding patents have already been granted in the United Kingdom, and the European Patent Office is also granting a patent to UC, which will issue on May 10, 2017. UC’s earliest patent application describing the CRISPR/Cas9 genome editing technology and its use was filed on May 25, 2012, while the Broad’s earliest patent application was filed more than six months later, on December 12, 2012.

The law firm of Munger, Tolles & Olson LLP will be handling the appeal, with Don Verrilli, former Solicitor General of the United States, as lead counsel.



Editas’ rivals appeal a recent setback on patent fight, mapping a global war for CRISPR supremacy

They say they are “pursuing applications in the U.S. and other jurisdictions worldwide to obtain patents claiming the CRISPR/Cas9 technology and its use in non-cellular and cellular settings, including eukaryotic cells. Corresponding patents have already been granted in the United Kingdom, and the European Patent Office is also granting a patent to UC, which will issue on May 10, 2017. UC’s earliest patent application describing the CRISPR/Cas9 genome editing technology and its use was filed on May 25, 2012, while the Broad’s earliest patent application was filed more than six months later, on December 12, 2012.”

The group said today it is also waging a global patent battle for CRISPR/Cas9 supremacy over Editas and its scientific founder, Feng Zhang, who patented the rival technology at The Broad.




Other press releases by Intellia Therapeutics, Inc.

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Reporter and Curator: Dr. Sudipta Saha, Ph.D.


Scientists think excessive population growth is a cause of scarcity and environmental degradation. A male pill could reduce the number of unintended pregnancies, which accounts for 40 percent of all pregnancies worldwide.


But, big drug companies long ago dropped out of the search for a male contraceptive pill which is able to chemically intercept millions of sperm before they reach a woman’s egg. Right now the chemical burden for contraception relies solely on the female. There’s not much activity in the male contraception field because an effective solution is available on the female side.


Presently, male contraception means a condom or a vasectomy. But researchers from Center for Drug Discovery at Baylor College of Medicine, USA are renewing the search for a better option—an easy-to-take pill that’s safe, fast-acting, and reversible.


The scientists began with lists of genes active in the testes for sperm production and motility and then created knockout mice that lack those genes. Using the gene-editing technology called CRISPR, in collaboration with Japanese scientists, they have so far made more than 75 of these “knockout” mice.


They allowed these mice to mate with normal (wild type) female mice, and if their female partners don’t get pregnant after three to six months, it means the gene might be a target for a contraceptive. Out of 2300 genes that are particularly active in the testes of mice, the researchers have identified 30 genes whose deletion makes the male infertile. Next the scientists are planning a novel screening approach to test whether any of about two billion chemicals can disable these genes in a test tube. Promising chemicals could then be fed to male mice to see if they cause infertility.


Female birth control pills use hormones to inhibit a woman’s ovaries from releasing eggs. But hormones have side effects like weight gain, mood changes, and headaches. A trial of one male contraceptive hormone was stopped early in 2011 after one participant committed suicide and others reported depression. Moreover, some drug candidates have made animals permanently sterile which is not the goal of the research. The challenge is to prevent sperm being made without permanently sterilizing the individual.


As a better way to test drugs, Scientists at University of Georgia, USA are investigating yet another high-tech approach. They are turning human skin cells into stem cells that look and act like the spermatogonial cells in the testes. Testing drugs on such cells might provide more accurate leads than tests on mice.


The male pill would also have to start working quickly, a lot sooner than the female pill, which takes about a week to function. Scientists from University of Dundee, U.K. admitted that there are lots of challenges. Because, a women’s ovary usually release one mature egg each month, while a man makes millions of sperm every day. So, the male pill has to be made 100 percent effective and act instantaneously.



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CRISPR Patent Battle Determined on 2/15/2017 – USPTO issues a verdict in legal tussle over rights to genome-editing technology

Curator: Aviva Lev-Ari, PhD, RN

Broad Institute prevails in heated dispute over CRISPR patents

Sharon Begley

In a one-sentence judgment by the Patent Trial and Appeal Board, the three judges decided that there is “no interference in fact.” In other words, key CRISPR patents awarded to the Broad beginning in 2014 are sufficiently different from patents applied for by UC that they can stand. The judges’ full 51-page decision explaining their reasoning stated that the Broad had persuaded them “that the parties claim patentably distinct subject matter.”


The Broad said in a statement that the decision “confirms that the patents and applications of Broad Institute and UC Berkeley are about different subjects and do not interfere with each other.”

UC, Berkeley

In a statement, the University of California said it was pleased that its patent application, which it described as covering “the invention and use of CRISPR gene editing in all cells,” can move forward. “We continue to maintain that the evidence overwhelmingly supports our position that the Doudna/Charpentier team was the first group to invent this technology for use in all settings and all cell types,” it said, “and that the Broad Institute’s patents directed toward use of the CRISPR-Cas9 system in particular cell types are not patentably distinct from the Doudna/Charpentier invention.”

UC said it is considering its legal options, including the possibility of an appeal, but it contended that anyone who wants to develop CRISPR-based treatments for human diseases would have to license not only the Broad’s patents but also those that UC expects to be awarded. “Ours,” Doudna told reporters, “is for the use [of CRISPR] in all cells,” including human ones.

PTAB appeals are heard by the US Court of Appeals for the Federal Circuit, which sits in Washington. In recent years, more than half of PTAB’s decisions have been upheld.

“The Federal Circuit heard three appeals of interferences in 2016,” said Sherkow. “All three were at least affirmed in part. It’s completely unclear whether that’s meaningful — it’s an N of 3–but there you go.” Overall, on 155 appeals since PTAB was created in 2012, the Federal Circuit affirmed 120 on every issue, dismissed or reversed 21 on every issue, and issued partial decisions (that is, upholding parts of a PTAB decision and reversing others) in the other 14.

Said UC attorney Lynn Pasahow:

For “all tennis balls,” read “all cells.” For “green tennis balls,” read “eukaryotic cells.”


What will that mean for licensees of CRISPR patents?

Stanford University Voice

UC believes that any company that wants to use CRISPR to develop human therapies — we’re looking at you, Editas Medicine — will need to license not only the Broad’s patents on eukaryotic cells but also those UC expects to receive on all kinds of cells. “It looks to me as if someone wanting to use the Broad patent would also have to license the UC patent,” agreed law professor Hank Greely of Stanford University. “The UC patent (if granted) would be on any use; the Broad would be on use in eukaryotes. I think someone who wanted to do this in eukaryotes would need to have licenses to both.”

CRISPR-Cas9 is unlikely to be the last genome-editing technology ever discovered. In 2015, Zhang and his colleagues discovered a version called Cpf1, which they’ve now patented and licensed to Editas. “I continue to think the possibility of inventing around the [CRISPR] patents seems very likely,” said Stanford’s Greely. Bacteria “have certainly come up with other ways to reach the same end [of genome editing], ways that aren’t covered by UC’s or the Broad’s claims. That could make either of these patents ultimately of little importance … especially if the licensing conditions give people a strong incentive to come up with invent-arounds.” Science will march on.

What does the CRISPR ruling mean for biotech?

By DAMIAN GARDE @damiangarde

FEBRUARY 15, 2017

Editas Medicine, which has aligned with the winning Broad, saw its share price rise more than 25 percent on Wednesday. Intellia Therapeutics, affiliated with UC, fell about 11 percent, while compatriot CRISPR Therapeutics dipped 24 percent.


Broad Institute wins bitter battle over CRISPR patents

The US Patent and Trademark Office issues a verdict in legal tussle over rights to genome-editing technology.

15 February 2017 Updated:In December 2016, lawyers representing the University of California and the Broad Institute participated in oral arguments before a trio of patent-court judges. University of California attorney Lynn Pasahow said that the team had not yet decided whether it would appeal the verdict on 2/15/2017.

Lawyers representing the University of California filed for an ‘interference’ proceeding, in an effort to have the Broad’s patents thrown out. But on 15 February, patent judges determined that there was no interference, meaning that the Broad’s invention is distinct from that of the University of California, and the Broad patents will stand. The University of California’s patent application will now be referred back to an examiner, but legal challenges could continue.

molecular biologist Jennifer Doudna of the University of California in Berkeley, likened the situation to licensing permission to someone who wants to use green tennis balls. “They will have a patent on the green tennis balls,” she said, referring to the Broad patents. “We will have a patent on all tennis balls.” ”Doudna argued at the press conference that the patent battle had not hampered research, given the speed with which researchers had taken up the technique and companies had rushed to commercialize it.”

The University of California’s invention would cover the design of the RNA molecule that guides the key step in CRISPR–Cas9 gene editing, directing the Cas9 enzyme to a specific site in the genome. But getting that system to work in eukaryotes was an additional inventive step, Coombes says, a patent lawyer at intellectual-property specialists HGF in York, UK.


Nature doi:10.1038/nature.2017.21502

Related articles from

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

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


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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


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

See List, below


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




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



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.



Barrangou, R. (2012). RNA-mediated programmable DNA cleavage. Nat. Biotechnol. 30, 836–838. Barrangou, R., and Marraffini, L.A. (2014). CRISPR-Cas systems: Prokaryotes upgrade to adaptive immunity. Mol. Cell 54, 234–244. Barrangou, R., Fremaux, C., Deveau, H., Richards, M., Boyaval, P., Moineau, S., Romero, D.A., and Horvath, P. (2007). CRISPR provides acquired resistance against viruses in prokaryotes. Science 315, 1709–1712. Bibikova, M., Carroll, D., Segal, D.J., Trautman, J.K., Smith, J., Kim, Y.G., and Chandrasegaran, S. (2001). Stimulation of homologous recombination through targeted cleavage by chimeric nucleases. Mol. Cell. Biol. 21, 289–297. 26 Cell 164, January 14, 2016 ª2016 Elsevier Inc. Bibikova, M., Beumer, K., Trautman, J.K., and Carroll, D. (2003). Enhancing gene targeting with designed zinc finger nucleases. Science 300, 764. Boch, J., Scholze, H., Schornack, S., Landgraf, A., Hahn, S., Kay, S., Lahaye, T., Nickstadt, A., and Bonas, U. (2009). Breaking the code of DNA binding specificity of TAL-type III effectors. Science 326, 1509–1512. Bolotin, A., Quinquis, B., Sorokin, A., and Ehrlich, S.D. (2005). Clustered regularly interspaced short palindrome repeats (CRISPRs) have spacers of extrachromosomal origin. Microbiology 151, 2551–2561. Brouns, S.J.J., Jore, M.M., Lundgren, M., Westra, E.R., Slijkhuis, R.J.H., Snijders, A.P.L., Dickman, M.J., Makarova, K.S., Koonin, E.V., and van der Oost, J. (2008). Small CRISPR RNAs guide antiviral defense in prokaryotes. Science 321, 960–964. Capecchi, M.R. (2005). Gene targeting in mice: functional analysis of the mammalian genome for the twenty-first century. Nat. Rev. Genet. 6, 507–512. Carroll, D. (2012). A CRISPR approach to gene targeting. Mol. Ther. 20, 1658– 1660. Cho, S.W., Kim, S., Kim, J.M., and Kim, J.-S. (2013). Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease. Nat. Biotechnol. 31, 230–232. Cong, L., Ran, F.A., Cox, D., Lin, S., Barretto, R., Habib, N., Hsu, P.D., Wu, X., Jiang, W., Marraffini, L.A., and Zhang, F. (2013). Multiplex genome engineering using CRISPR/Cas systems. Science 339, 819–823. Deltcheva, E., Chylinski, K., Sharma, C.M., Gonzales, K., Chao, Y., Pirzada, Z.A., Eckert, M.R., Vogel, J., and Charpentier, E. (2011). CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III. Nature 471, 602–607. Deveau, H., Barrangou, R., Garneau, J.E., Labonte´ , J., Fremaux, C., Boyaval, P., Romero, D.A., Horvath, P., and Moineau, S. (2008). Phage response to CRISPR-encoded resistance in Streptococcus thermophilus. J. Bacteriol. 190, 1390–1400. Garneau, J.E., Dupuis, M.-E` ., Villion, M., Romero, D.A., Barrangou, R., Boyaval, P., Fremaux, C., Horvath, P., Magada´ n, A.H., and Moineau, S. (2010). The CRISPR/Cas bacterial immune system cleaves bacteriophage and plasmid DNA. Nature 468, 67–71. Gasiunas, G., Barrangou, R., Horvath, P., and Siksnys, V. (2012). Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria. Proc. Natl. Acad. Sci. USA 109, E2579–E2586. Haber, J.E. (2000). Lucky breaks: analysis of recombination in Saccharomyces. Mutat. Res. 451, 53–69. Hale, C.R., Zhao, P., Olson, S., Duff, M.O., Graveley, B.R., Wells, L., Terns, R.M., and Terns, M.P. (2009). RNA-guided RNA cleavage by a CRISPR RNA-Cas protein complex. Cell 139, 945–956. Horvath, P., Romero, D.A., Couˆ te´-Monvoisin, A.-C., Richards, M., Deveau, H., Moineau, S., Boyaval, P., Fremaux, C., and Barrangou, R. (2008). Diversity, activity, and evolution of CRISPR loci in Streptococcus thermophilus. J. Bacteriol. 190, 1401–1412. Hsu, P.D., Lander, E.S., and Zhang, F. (2014). Development and applications of CRISPR-Cas9 for genome engineering. Cell 157, 1262–1278. Hwang, W.Y., Fu, Y., Reyon, D., Maeder, M.L., Tsai, S.Q., Sander, J.D., Peterson, R.T., Yeh, J.R., and Joung, J.K. (2013). Efficient genome editing in zebrafish using a CRISPR-Cas system. Nat. Biotechnol. 31, 227–229. Ishino, Y., Shinagawa, H., Makino, K., Amemura, M., and Nakata, A. (1987). Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product. J. Bacteriol. 169, 5429–5433. Jansen, R., Embden, J.D.A.V., Gaastra, W., and Schouls, L.M. (2002). Identifi- cation of genes that are associated with DNA repeats in prokaryotes. Mol. Microbiol. 43, 1565–1575. Jasin, M., and Rothstein, R. (2013). Repair of strand breaks by homologous recombination. Cold Spring Harb. Perspect. Biol. 5, a012740. Jiang, W., and Marraffini, L.A. (2015). CRISPR-Cas: New tools for genetic manipulations from bacterial immunity systems. Annu. Rev. Microbiol. 69, 209–228. Jinek, M., Chylinski, K., Fonfara, I., Hauer, M., Doudna, J.A., and Charpentier, E. (2012). A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 337, 816–821. Jinek, M., East, A., Cheng, A., Lin, S., Ma, E., and Doudna, J. (2013). RNA-programmed genome editing in human cells. eLife 2, e00471. Makarova, K.S., Grishin, N.V., Shabalina, S.A., Wolf, Y.I., and Koonin, E.V. (2006). A putative RNA-interference-based immune system in prokaryotes: computational analysis of the predicted enzymatic machinery, functional analogies with eukaryotic RNAi, and hypothetical mechanisms of action. Biol. Direct 1, 7. Makarova, K.S., Wolf, Y.I., Alkhnbashi, O.S., Costa, F., Shah, S.A., Saunders, S.J., Barrangou, R., Brouns, S.J.J., Charpentier, E., Haft, D.H., et al. (2015). An updated evolutionary classification of CRISPR-Cas systems. Nat. Rev. Microbiol. 13, 722–736. Mali, P., Yang, L., Esvelt, K.M., Aach, J., Guell, M., DiCarlo, J.E., Norville, J.E., and Church, G.M. (2013). RNA-guided human genome engineering via Cas9. Science 339, 823–826. Mangold, M., Siller, M., Roppenser, B., Vlaminckx, B.J.M., Penfound, T.A., Klein, R., Novak, R., Novick, R.P., and Charpentier, E. (2004). Synthesis of group A streptococcal virulence factors is controlled by a regulatory RNA molecule. Mol. Microbiol. 53, 1515–1527. Marraffini, L.A., and Sontheimer, E.J. (2008). CRISPR interference limits horizontal gene transfer in staphylococci by targeting DNA. Science 322, 1843–1845. Medawar, P. (1968). Lucky Jim (New York Review of Books), March 28, 1968. Miller, J.C., Tan, S., Qiao, G., Barlow, K.A., Wang, J., Xia, D.F., Meng, X., Paschon, D.E., Leung, E., Hinkley, S.J., et al. (2011). A TALE nuclease architecture for efficient genome editing. Nat. Biotechnol. 29, 143–148. Mojica, F.J.M., and Garrett, R.A. (2012). Discovery and Seminal Developments in the CRISPR Field. In CRISPR-Cas Systems, R. Barrangou and J. van der Oost, eds. (Berlin, Heidelberg: Springer Berlin Heidelberg), pp. 1–31. Mojica, F.J.M., Juez, G., and Rodrı´guez-Valera, F. (1993). Transcription at different salinities of Haloferax mediterranei sequences adjacent to partially modified PstI sites. Mol. Microbiol. 9, 613–621. Mojica, F.J.M., Ferrer, C., Juez, G., and Rodrı´guez-Valera, F. (1995). Long stretches of short tandem repeats are present in the largest replicons of the Archaea Haloferax mediterranei and Haloferax volcanii and could be involved in replicon partitioning. Mol. Microbiol. 17, 85–93. Mojica, F.J.M., Dı´ez-Villasen˜ or, C., Soria, E., and Juez, G. (2000). Biological significance of a family of regularly spaced repeats in the genomes of Archaea, Bacteria and mitochondria. Mol. Microbiol. 36, 244–246. Mojica, F.J.M., Dı´ez-Villasen˜ or, C., Garcı´a-Martı´nez, J., and Soria, E. (2005). Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements. J. Mol. Evol. 60, 174–182. Moscou, M.J., and Bogdanove, A.J. (2009). A simple cipher governs DNA recognition by TAL effectors. Science 326, 1501–1501. Pandika, M. (2014) Jennifer Doudna, CRISPR Code Killer,, January 7, 2014. < >. Porteus, M.H., and Baltimore, D. (2003). Chimeric nucleases stimulate gene targeting in human cells. Science 300, 763. Pourcel, C., Salvignol, G., and Vergnaud, G. (2005). CRISPR elements in Yersinia pestis acquire new repeats by preferential uptake of bacteriophage DNA, and provide additional tools for evolutionary studies. Microbiology 151, 653–663. Sander, J.D., and Joung, J.K. (2014). CRISPR-Cas systems for editing, regulating and targeting genomes. Nat. Biotechnol. 32, 347–355. Cell 164, January 14, 2016 ª2016 Elsevier Inc. 27 Sapranauskas, R., Gasiunas, G., Fremaux, C., Barrangou, R., Horvath, P., and Siksnys, V. (2011). The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli. Nucleic Acids Res. 39, 9275–9282. Sharma, C.M., Hoffmann, S., Darfeuille, F., Reignier, J., Findeiss, S., Sittka, A., Chabas, S., Reiche, K., Hackermu¨ ller, J., Reinhardt, R., et al. (2010). The primary transcriptome of the major human pathogen Helicobacter pylori. Nature 464, 250–255. Siksnys, V., Gasiunas, G., and Karvelis, T. (2012). RNA-directed DNA cleavage by the Cas9-crRNA complex from CRISPR3/Cas immune system of Streptococcus thermophilus. U.S. Provisional Patent Application 61/613,373, filed March 20, 2012; later published as US2015/0045546 (pending). Sontheimer, E., and Marraffini, L. (2008). Target DNA interference with crRNA. U.S. Provisional Patent Application 61/009,317, filed September 23, 2008; later published as US2010/0076057 (abandoned). Sorek, R., Kunin, V., and Hugenholtz, P. (2008). CRISPR–a widespread system that provides acquired resistance against phages in bacteria and archaea. Nat. Rev. Microbiol. 6, 181–186. Sternberg, S.H., and Doudna, J.A. (2015). Expanding the Biologist’s Toolkit with CRISPR-Cas9. Mol. Cell 58, 568–574. Travis, J. (2015). GENETIC ENGINEERING. Germline editing dominates DNA summit. Science 350, 1299–1300. Urnov, F.D., Miller, J.C., Lee, Y.-L., Beausejour, C.M., Rock, J.M., Augustus, S., Jamieson, A.C., Porteusm, M.H., Gregory, P.D., and Holmes, M.C. (2005). Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature 435, 646–651. van der Oost, J., Westra, E.R., Jackson, R.N., and Wiedenheft, B. (2014). Unravelling the structural and mechanistic basis of CRISPR-Cas systems. Nat. Rev. Microbiol. 12, 479–492. Wright, A.V., James, K., Nun˜ ez, J.K., and Doudna, J.A. (2016). Biology and applications of CRISPR systems: Harnessing nature’s toolbox for genome engineering. Cell 164, this issue, 29–44. Zetsche, B., Gootenberg, J.S., Abudayyeh, O.O., Slaymaker, I.M., Makarova, K.S., Essletzbichler, P., Volz, S.E., Joung, J., van der Oost, J., Regev, A., et al. (2015). Cpf1 Is aSingleRNA-Guided Endonuclease of a Class 2 CRISPR-Cas System. Cell. Zhang, F., Cong, Le, Lodato, S., Kosuri, S., Church, G.M., and Arlotta, P. (2011). Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription. Nat. Biotechnol. 29, 149–153. Zhang, F. (2012). Systems Methods and Compositions for Sequence Manipulation. U.S. Provisional Patent Application 61/736,527, filed December 12, 2012; later published as US008697359B1 (awarded)

Top 50 Women in CRISPR : Women in CRISPR

Women in CRISPR/Cas9 genome editing research – List Version 3
First Name
Last Name Organisation Location Country Position Website
Twitter Handle
Field of Research
Research Interest
Divaki Bhaya Stanford Univeristy Stanford, CA USA Professor
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.
Jill Banfield University of California Berkeley Berkeley, CA USA Professor 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.
Denis Bauer
Commonwealth Scientific and Industrial Research Organisation (CSIRO)
Sydney Australia Head of laboratory @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.
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.
Alexa Burger University of Zurich Zurich Switzerland
Senior postdoctoral fellow @aburger2009 Zebrafish – Technology development
CRISPR application in Zebrafish (ribonucleic complex and increase mutation efficiency)
Emmanuelle Charpentier Max Plank Institute Berlin Germany Professor 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.
Sylvia Comporesi Kings College London London UK Lecturer @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.
Elena Conti Max Plank Institute Martinsried Germany
Group leader and Director 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.
Jennifer Doudna University of California Berkeley Berkeley, CA USA Professor @doudna_lab RNA biology – Adaptive immunity
Exploring molecular mechanisms of RNA-mediated gene regulation
Caixia Gao Chinese Academy of Science Beijing China Professor 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.
Carine Giovanangeli Museum National d’Histoire Naturelle Paris France Director of Researchénomiques+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).
Natalia Gomez-Ospina Stanford Univeristy Stanford, CA USA Clinical Instructor 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.
Asma Hatoum-Aslan The University of Alabama Tuscaloosa, AL USA Assistant Professor @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.
Rachel Haurwitz Caribou Biosciences Berkeley, CA USA
President and Chief Executive officer 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.
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.
Nina Hoyland-kroghsbo Princeton University Princeton, NJ USA Postdoctoral fellow 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.
Danwei Huangfu Memorial Sloan Kettering New York, NY USA Head of laboratory 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.
Maria Jasin Memorial Sloan Kettering New York, NY USA Head of laboratory 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
Josephin Johnston The Hasting Centre Garrison, NY USA Director of Research @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.
Helene Jousset-Sabroux The Walter and Eliza Hall Institute for Medical Research Melbourne Australia Head of laboratoryélène-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.
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.
Hong Li Florida State University Tallahassee, FL USA Professor 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.
Jennifer Listgarten Microsoft Research Cambridge, MA USA Senior Researcher 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.
Shirley Liu Dana Farber Cancer Institute – Harvard Cambridge, MA USA Head of laboratory 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.
Anita Marchfelder Ulm University Ulm Germany Head of laboratory 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.
Karen Maxwell University of Toronto Toronto Canada Assistant Professor @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.
Barbara J Meyer University of California Berkeley Berkeley, CA USA Head of laboratory 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.
Shondra Miller Washington University St Louis, MO USA Director of Research 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.
Hiromi Miura Tokai University School of Medicine Kanagawa Japan Assistant Professor Mouse – Technology development
Kathy Niakan The Francis Crick Institute London UK Head of laboratory 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.
Kate O’Connor-Giles University Wisconson Madison Madison, WI USA Head of laboratory 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.
April Pawluk University of California Berkeley Berkeley, CA USA Postdoctoral fellow @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.
Jennifer Phillips University of Oregon Eugene, OR USA Research Fellow @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.
Wenning Qin Biogen inc Cambridge, MA USA Director of Research @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.
Rakhi Rajan The University of Oklahoma Norman, OK USA Assistant Professor 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.
Dipali Sashital Iowa State University Ames, IA USA Assistant Professor @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.
Nikki Shariat Gettysburg College Gettysburg, PA USA Assistant Professor 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.
Bettina Schmid Deutsches Zentrum fur Neurodegenerative Erkankungen Helmotz Germany Head of laboratory 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).
Kimberley Seed University of California Berkeley Berkeley, CA USA Assistant Professor 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.
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).
Joyce Van Eyck Cornell Univeristy Ithaca, NY USA Assistant Professor 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.
Stineke Van Houte University of Exeter Exeter UK Research Fellow 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.
Leslie Vosshall The Rockfeller Univeristy New York, NY USA Head of laboratory @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.
Kan Wang Iowa State University Agron,IA USA Professor 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.
Rachel Whitaker University of Illinois at Urbana Champaign Urbana, IL USA Associate Professor 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.
Susan Woods University of Adelaide Adelaide Australia Senior Research Fellow 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.
Luhan Yang eGenesis Cambridge, MA USA Co-founder and CSO 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.
Yan Zhang University of Michigan Ann Arbor, MI USA Assistant Professor






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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Dr. Doudna: RNA synthesis capabilities of Synthego’s team represent a significant leap forward for Synthetic Biology

Reporter: Aviva Lev-Ari, PhD, RN


Synthego Raises $41 Million From Investors, Including a Top Biochemist

Synthego also drew in Dr. Doudna, who had crossed paths with the company’s head of synthetic biology at various industry conferences. According to Mr. Dabrowski, the money from her trust represents the single-biggest check from a non-institutional investor that the start-up has raised.

Synthego’s new funds will help the company take its products to a more global customer base, as well as broaden its offerings. The longer-term goal, Mr. Dabrowski said, is to help fully automate biotech research and take care of much of the laboratory work that scientists currently handle themselves.

The model is cloud technology, where companies rent out powerful remote server farms to handle their computing needs rather than rely on their own hardware.

“We’ll be able to do their full research workflow,” he said. “If you look at how cloud computing developed, it used to be that every company handled their server farm. Now it’s all handled in the cloud.”


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

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


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