In vivo gene editing in dystrophic mouse muscle and muscle stem cells.

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][1], p. [403][2], p. [407][3] [1]: /lookup/doi/10.1126/science.aad5725 [2]: /lookup/doi/10.1126/science.aad5143 [3]: /lookup/doi/10.1126/science.aad5177