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Prevention of muscular dystrophy in mice by CRISPR/Cas9–mediated editing of germline DNA

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Science  05 Sep 2014:
Vol. 345, Issue 6201, pp. 1184-1188
DOI: 10.1126/science.1254445

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Genome editing corrects a muscle disease

Patients with Duchenne muscular dystrophy find their muscles growing progressively weaker. Studies identified dystrophin as the culprit gene, which galvanized research into gene-targeted therapies. Long et al. apply genome editing to “correct” the disease-causing mutation in mice genetically destined to develop the disease. This germline editing strategy kept muscles from degenerating, even in mice harboring only a small percentage of corrected cells. Although not feasible for humans, this proof of concept sets the stage for applying genome editing to specific cell types involved in the disease.

Science, this issue p. 1184