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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Detaylı Bibliyografya
Yayımlandı:Mol Ther
Asıl Yazarlar: Ousterout, David G, Kabadi, Ami M, Thakore, Pratiksha I, Perez-Pinera, Pablo, Brown, Matthew T, Majoros, William H, Reddy, Timothy E, Gersbach, Charles A
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Nature Publishing Group 2015
Konular:
Original Article
Online Erişim: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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