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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2015
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4351462/
https://www.ncbi.nlm.nih.gov/pubmed/25492562
http://dx.doi.org/10.1038/mt.2014.234

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4351462/
https://www.ncbi.nlm.nih.gov/pubmed/25492562
http://dx.doi.org/10.1038/mt.2014.234

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