Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping

Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder caused by mutations in the dystrophin gene that result in the absence of functional protein. Antisense-mediated exon-skipping is one of the most promising approaches for the treatment of DMD because of its capacity to correct the r...

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Bibliografske podrobnosti
Main Authors: Goyenvalle, Aurélie, Babbs, Arran, Powell, Dave, Kole, Ryszard, Fletcher, Sue, Wilton, Steve D, Davies, Kay E
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2009
Teme:
Original Articles
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC2839228/
https://ncbi.nlm.nih.gov/pubmed/19844193
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.248
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