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CRISPR alleviates muscular dystrophy in dogs

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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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Authors and Affiliations

  1. Departments of Molecular Microbiology and Immunology, Neurology, Bioengineering and Biomedical Sciences, University of Missouri, Columbia, MO, USA

    Dongsheng Duan

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  1. Dongsheng Duan
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Correspondence to Dongsheng Duan.

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Competing interests

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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