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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2013
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3776627/
https://www.ncbi.nlm.nih.gov/pubmed/23732986
http://dx.doi.org/10.1038/mt.2013.111

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3776627/
https://www.ncbi.nlm.nih.gov/pubmed/23732986
http://dx.doi.org/10.1038/mt.2013.111

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