Safety concerns surrounding AAV and CRISPR therapies in neuromuscular treatment.
Med (New York, N.Y.), 4(12): 855-856 PMID 38070478 DOI 10.1016/j.medj.2023.11.008
Abstract
Gene therapies, notably those leveraging CRISPR and adeno-associated virus vectors (AAVs), have risen to the forefront of potential treatments for neuromuscular disorders. The recent demise of a Duchenne muscular dystrophy (DMD) patient following a trial utilizing CRISPR transactivation with AAV has cast a spotlight on the potential risks associated with these approaches.1.