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Surrogate gene therapy for muscular dystrophy

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An engineered truncated gene derived from the dystrophin-related protein (utrophin), prevents pathology without an immune response in an animal model of Duchenne muscular dystrophy (DMD) gene therapy.

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Fig. 1: Utrophin gene therapy for DMD.

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Authors and Affiliations

  1. MDUK Oxford Neuromuscular Centre, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK

    Kay E. Davies

  2. University of Washington School of Medicine, Departments of Neurology, Medicine and Biochemistry, Wellstone Muscular Dystrophy Specialized Research Center, Seattle, Washington, USA

    Jeffrey S. Chamberlain

Authors
  1. Kay E. Davies
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  2. Jeffrey S. Chamberlain
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Correspondence to Kay E. Davies.

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Competing interests

J.S.C. is a member of the Scientific Advisory Board for Solid Biosciences. K.E.D. has no declared conflicts.

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Davies, K.E., Chamberlain, J.S. Surrogate gene therapy for muscular dystrophy. Nat Med 25, 1473–1474 (2019). https://doi.org/10.1038/s41591-019-0604-2

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