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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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Hlavní autoři: Brolin, Camilla, Shiraishi, Takehiko
Médium: Artigo
Jazyk:Inglês
Vydáno: Landes Bioscience 2011
Témata:
On-line přístup: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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