Safety concerns surrounding AAV and CRISPR therapies in neuromuscular treatment

Harry Wilton-Clark; Toshifumi Yokota

doi:10.1016/j.medj.2023.11.008

Safety concerns surrounding AAV and CRISPR therapies in neuromuscular treatment

Med. 2023 Dec 8;4(12):855-856. doi: 10.1016/j.medj.2023.11.008.

Authors

Harry Wilton-Clark¹, Toshifumi Yokota²

Affiliations

¹ University of Alberta, Department of Medical Genetics, Edmonton, AB, Canada.
² University of Alberta, Department of Medical Genetics, Edmonton, AB, Canada. Electronic address: toshifum@ualberta.ca.

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.

MeSH terms

Clustered Regularly Interspaced Short Palindromic Repeats* / genetics
Dependovirus / genetics
Genetic Therapy / adverse effects
Genetic Vectors / adverse effects
Genetic Vectors / genetics
Humans
Muscular Dystrophy, Duchenne* / genetics
Muscular Dystrophy, Duchenne* / therapy