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With 1 Cure Behind It, This Breakthrough Biotech Takes Aim at a Bigger Market

May 7, 2021 at 6:40AM Long before they won the 2020 Nobel Prize in Chemistry for discovering the molecular gene-editing tool known as clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9, Jennifer Doudna and Emmanuelle Charpentier dreamed of Victoria Gray, a woman with sickle cell disease. Although they didn t know her, they knew that one day, a patient with a genetic disease would be cured using their discovery.  Sickle cell disease was the logical place to start since it s the most common blood disorder with a single-gene mutation. CRISPR Therapeutics (NASDAQ:CRSP) is one of several companies using gene editing to fight the illness. Their drug CTX001, developed with partner

CRISPR s Rapid Rise Shakes Up Genome-Wide Screening

CRISPR’s Rapid Rise Shakes Up Genome-Wide Screening Source: Vince Dittmer/NYGC CRISPR’s Rapid Rise Shakes Up Genome-Wide Screening A truly disruptive technology, CRISPR screening roughly displaces its predecessors then refines itself, as shown by its functional genomics applications and its ability to complement single-cell transcriptomics May 3, 2021 Using a genome-scale, loss-of-function CRISPR screen, scientists from New York University, the New York Genome Center, and the Icahn School of Medicine at Mount Sinai identified human genes and gene regulatory networks that are required by SARS-CoV-2. The scientists also demonstrated that suppression of these genes and networks confers resistance to viral infection. For example, loss of RAB7A effectively sequesters the ACE2 receptor inside cells, preventing it from serving as an entry point for the virus. [Vince Dittmer/NYGC]

PICTURES: Your fabulous furry five- Dorset s cutest pets

PICTURES: Your fabulous furry five- Dorset s cutest pets
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