Evaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a severe muscle wasting disorder typically caused by frame-shifting mutations in the DMD gene. Restoration of the reading frame would allow the production of a shorter but partly functional dystrophin protein as seen in Becker muscular dystrophy patients. This ca...

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Bibliographic Details
Published in:Mol Ther Nucleic Acids
Main Authors: Jirka, Silvana M G, Tanganyika-de Winter, Christa L, Boertje-van der Meulen, Joke W, van Putten, Maaike, Hiller, Monika, Vermue, Rick, de Visser, Peter C, Aartsma-Rus, Annemieke
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2015
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Original Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC5014533/
https://ncbi.nlm.nih.gov/pubmed/26623937
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2015.39
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