In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

TLDR

CRISPR/Cas9 gene‑editing offers a potential therapeutic approach for muscular dystrophy by correcting the dystrophin gene in somatic cells, despite ethical debates over germline editing. The study delivered CRISPR/Cas9 via adeno‑associated virus‑9 to young dystrophin‑mutant mice. Partial restoration of dystrophin in skeletal and cardiac muscle led to improved skeletal muscle function. Published in Science, pages 400, 403, and 407.

Abstract

Editing can help build stronger muscles Much of the controversy surrounding the gene-editing technology called CRISPR/Cas9 centers on the ethics of germline editing of human embryos to correct disease-causing mutations. For certain disorders such as muscular dystrophy, it may be possible to achieve therapeutic benefit by editing the faulty gene in somatic cells. In proof-of-concept studies, Long et al. , Nelson et al. , and Tabebordbar et al. used adeno-associated virus-9 to deliver the CRISPR/Cas9 gene-editing system to young mice with a mutation in the gene coding for dystrophin, a muscle protein deficient in patients with Duchenne muscular dystrophy. Gene editing partially restored dystrophin protein expression in skeletal and cardiac muscle and improved skeletal muscle function. Science , this issue p. 400 , p. 403 , p. 407