Correction of Three Prominent Mutations in Mouse and Human Models of Duchenne Muscular Dystrophy by Single-Cut Genome Editing

Duchenne muscular dystrophy (DMD), one of the most common neuromuscular disorders of children, is caused by the absence of dystrophin protein in striated muscle. Deletions of exons 43, 45, and 52 represent mutational “hotspot” regions in the dystrophin gene. We created three new DMD mouse models har...

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Foilsithe in:Mol Ther
Main Authors: Min, Yi-Li, Chemello, Francesco, Li, Hui, Rodriguez-Caycedo, Cristina, Sanchez-Ortiz, Efrain, Mireault, Alex A., McAnally, John R., Shelton, John M., Zhang, Yu, Bassel-Duby, Rhonda, Olson, Eric N.
Formáid: Artigo
Teanga:Inglês
Foilsithe: American Society of Gene & Cell Therapy 2020
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Original Article
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC7474267/
https://ncbi.nlm.nih.gov/pubmed/32892813
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.05.024
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