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Antisense mediated exon skipping therapy for duchenne muscular dystrophy (DMD)

Duchenne Muscular Dystrophy (DMD) is a lethal disease caused by mutations in the dystrophin gene (DMD) that result in the absence of essential muscle protein dystrophin. Among many different approaches for DMD treatment, exon skipping, mediated by antisense oligonucleotides, is one of the most promi...

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Bibliographic Details
Main Authors: Brolin, Camilla, Shiraishi, Takehiko
Format: Artigo
Language:Inglês
Published: Landes Bioscience 2011
Subjects:
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3116580/
https://ncbi.nlm.nih.gov/pubmed/21686247
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.4161/adna.2.1.15425
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