A Visual Guide to Gene Delivery
Codon
JULY 28, 2025
Casgevy , the first FDA-approved CRISPR-based therapy, is also an ex vivo gene therapy; it aims to cure sickle cell disease by editing a patient’s blood-producing stem cells so they generate functional fetal hemoglobin, then returning these corrected cells to the bloodstream. This changed with the discovery of AAVs.
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