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Cancer treatment using CRISPR-based Genome Editing System  Reporter: Irina Robu, PhD CRISPR, stands for “clusters of regularly interspaced short palindromic repeats” is one of the biggest accomplishments in science of this decade and it is the simplest tool for altering DNA sequences and modifying gene functions. The technology is adapted form the natural defense mechanism […]

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Novartis uses a ‘dimmer switch’ medication to fine-tune gene therapy candidates Reporter: Amandeep Kaur, BSc., MSc. Using viral vectors, lipid nanoparticles, and other technologies, significant progress has been achieved in refining the delivery of gene treatments. However, modifications to the cargo itself are still needed to increase safety and efficacy by better controlling gene expression. […]

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First single-course ‘curative’ CRISPR Shot by Intellia rivals Alnylam, Ionis and Pfizer Reporter: Aviva Lev-Ari, PhD, RN   UPDATED on 2/23/2023 As Novartis pulls back from legacy sickle cell program, Intellia CEO calls the market ‘wide open’ By Annalee ArmstrongFeb 23, 2023 10:10am Intellia announced in its fourth-quarter earnings report that Novartis had ended development of […]

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CRISPR-Cas9 and the Power of Butterfly Gene Editing Reporter: Madison Davis Genome editing is a relatively new branch of genetic engineering that utilizes modern technologies in altering, inserting, or deleting selective DNA sequences within cells.  CRISPR-Cas9, otherwise known as “Clustered Regularly Interspaced Short Palindromic Repeat”, is a groundbreaking genome editing technique for scientists, as it […]

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From AAAS Science News on COVID19: New CRISPR based diagnostic may shorten testing time to 5 minutes Reporter: Stephen J. Williams, Ph.D.                   A new CRISPR-based diagnostic could shorten wait times for coronavirus tests.     New test detects coronavirus in just 5 minutes By Robert F. ServiceOct. […]

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Prime Editing as a New CRISPR Tool to Enhance Precision and Versatility   Reporter: Stephen J. Williams, PhD   CRISPR has become a powerful molecular for the editing of genomes tool in research, drug discovery, and the clinic (see posts and ebook on this site below) Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene […]

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The Nobel Prize in Chemistry 2020: Emmanuelle Charpentier & Jennifer A. Doudna Reporters: Stephen J. Williams, Ph.D. and Aviva Lev-Ari, PhD, RN The Royal Swedish Academy of Sciences has decided to award the Nobel Prize in Chemistry 2020 to Emmanuelle CharpentierMax Planck Unit for the Science of Pathogens, Berlin, Germany Jennifer A. DoudnaUniversity of California, Berkeley, […]

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Live Conference Coverage AACR 2020 in Real Time: Monday June 22, 2020 Mid Day Sessions Reporter: Stephen J. Williams, PhD This post will be UPDATED during the next two days with notes from recordings from other talks Follow Live in Real Time using #AACR20 @pharma_BI @AACR               Register for […]

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Lessons on the Frontier of Gene & Cell Therapy – The Disruptive Dozen 12 #GCT Breakthroughs that are revolutionizing Healthcare Reporter: Aviva Lev-Ari, PhD, RN Mass General Brigham Innovation @MGBInnovation Read key takeaways from the 2022 World Medical Innovation Forum in this report from the Bank of America Institute. #WMIF2022 Quote Tweet Bank of America […]

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Genetic variation causes human lupus, systemic lupus erythematosus (SLE) Reporter: Aviva Lev-Ari, PhD, RN     TLR7 gain-of-function genetic variation causes human lupus Abstract Although circumstantial evidence supports enhanced Toll-like receptor 7 (TLR7) signalling as a mechanism of human systemic autoimmune disease1,2,3,4,5,6,7, evidence of lupus-causing TLR7 gene variants is lacking. Here we describe human systemic lupus erythematosus caused by […]

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