In the world of biopharmaceutical innovation, 2024 will be remembered as CRISPR’s breakout year. In the spring, five patients with sickle cell disease began treatment with Casgevy, the first FDA ...
View Full Profile. Learn about our Editorial Policies. “What’s significant about this is it’s taking CRISPR to that next step of what it can be used for, and in this case, it’s correcting mutations ...
CRISPR-Cas9 genome editing exploits the CRISPR-Cas system to modify a genome in a targeted manner. Guided by RNA, the Cas9 endonuclease breaks DNA at a target sequence. Imprecise repair of the ...