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

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
Main Authors: Silvana M G Jirka, Christa L Tanganyika-de Winter, Joke W Boertje-van der Meulen, Maaike van Putten, Monika Hiller, Rick Vermue, Peter C de Visser, Annemieke Aartsma-Rus
Format: Article
Language:English
Published: Elsevier 2015-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
antisense oligonucleotide
2’-deoxy-2’-fluoro
exon skipping
2’-O-methyl phosphorothioate
dystrophin
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253116300506

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http://www.sciencedirect.com/science/article/pii/S2162253116300506

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