Questions answered and unanswered by the first CRISPR editing study in the canine model of Duchenne muscular dystrophy.

Paradigms and Technologies
Methods Development

Abstract

CRISPR editing is being considered as a potential gene repair therapy to treat Duchenne muscular dystrophy (DMD), a dystrophin-deficient lethal muscle disease affecting all muscles in the body. A recent preliminary study from the Olson laboratory (Amoasii et al. 2018 Science 362:89-91) showed robust dystrophin restoration in a canine DMD model following intramuscular or intravenous delivery of the CRISPR editing machinery by adeno-associated virus serotype-9 (AAV9). Despite the limitation of the small sample size, short study duration and the lack of muscle function data, the Olson lab findings have provided important proof-of-principle for scaling up the CRISPR therapy from rodents to large mammals. Future large-scale, long-term, and comprehensive studies are warranted to establish the safety and efficacy of CRISPR editing therapy in large mammals.

Authors

Wasala, Nalinda B; Hakim, Chady; Chen, Shi-Jie; Yang, Nora; Duan, Dongsheng;

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