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GENOME ENGINEERING

CRISPR alleviates muscular dystrophy in dogs

Widespread editing of the mutated DMD gene by CRISPR–Cas9, systemically delivered via an adeno-associated virus, restores dystrophin expression in a canine model of Duchenne muscular dystrophy.

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Fig. 1: Scaling up body-wide gene editing via AAV-delivered CRISPR for restoring dystrophin levels in Duchenne muscular dystrophy.

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Correspondence to Dongsheng Duan.

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D.D. is a member of the scientific advisory board for Solid Biosciences and an equity holder of Solid Biosciences. D.D.’s lab has received research support from Solid Biosciences.

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Duan, D. CRISPR alleviates muscular dystrophy in dogs. Nat Biomed Eng 2, 795–796 (2018). https://doi.org/10.1038/s41551-018-0320-0

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