Cas9 is directed to its target within the DNA sequence through the use of the guide RNA. A specific sequence of DNA that is between two and five nucleotides in length must align with the 3’ end of the ...
Sickle cell disease is caused by a defect in the β-globin subunit of adult hemoglobin. Sickle hemoglobin polymerizes under hypoxic conditions, producing deformed red cells that hemolyze and cause vaso ...
The emergence of CRISPR-Cas9 genome-editing tools created new opportunities for enhancing plant and crop traits in agricultural biotechnology research. Now, multiplex genome-editing (MGE) technologies ...
Hereditary angioedema is a rare genetic disease that leads to severe and unpredictable swelling attacks. NTLA-2002 is an in vivo gene-editing therapy based on clustered regularly interspaced short ...
Expertise from Forbes Councils members, operated under license. Opinions expressed are those of the author. In the world of biopharmaceutical innovation, 2024 will be remembered as CRISPR’s breakout ...
Crispr Therapeutics, a leader in CRISPR/Cas9 biotechnology, recently received FDA and EMA approval for Casgevy, the first CRISPR/Cas9-based treatment for TDT and SCD. The partnership with Vertex ...
CHICAGO – A CRISPR-Cas9-based investigational therapy was linked with a drop in serum transthyretin (TTR) levels in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM), according to ...
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