Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients

Genome editing with engineered nucleases has recently emerged as an approach to correct genetic mutations by enhancing homologous recombination with a DNA repair template. However, many genetic diseases, such as Duchenne muscular dystrophy (DMD), can be treated simply by correcting a disrupted readi...

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Autors principals: Ousterout, David G, Perez-Pinera, Pablo, Thakore, Pratiksha I, Kabadi, Ami M, Brown, Matthew T, Qin, Xiaoxia, Fedrigo, Olivier, Mouly, Vincent, Tremblay, Jacques P, Gersbach, Charles A
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2013
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3776627/
https://ncbi.nlm.nih.gov/pubmed/23732986
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.111
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