Correction of Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Through Genomic Excision of Exon 51 by Zinc Finger Nucleases

Duchenne muscular dystrophy (DMD) is caused by genetic mutations that result in the absence of dystrophin protein expression. Oligonucleotide-induced exon skipping can restore the dystrophin reading frame and protein production. However, this requires continuous drug administration and may not gener...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Ousterout, David G, Kabadi, Ami M, Thakore, Pratiksha I, Perez-Pinera, Pablo, Brown, Matthew T, Majoros, William H, Reddy, Timothy E, Gersbach, Charles A
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2015
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4351462/
https://ncbi.nlm.nih.gov/pubmed/25492562
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.234
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