Melinda Kliegman

Jul 17, 2024 | nature.com | Melinda Kliegman |Jonathan H. Esensten |Ross Wilson |Fyodor Urnov

AbstractNineteen genetic therapies have been approved by the U.S. Food and Drug Administration (FDA) to date, a number that now includes the first CRISPR genome editing therapy for sickle cell disease, CASGEVY (exagamglogene autotemcel). This extraordinary milestone is widely celebrated because of the promise for future genome editing treatments of previously intractable genetic disorders and cancers.