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...

Descrición completa

Gardado en:
Detalles Bibliográficos
Publicado en: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: Nature Publishing Group 2015
Assuntos:
Original Article
Acceso en liña: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
Tags: Engadir etiqueta
Sen Etiquetas, Sexa o primeiro en etiquetar este rexistro!

Títulos similares