Long-term Exon Skipping Studies With 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models

Long-term Exon Skipping Studies With 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models

Antisense-mediated exon skipping for Duchenne muscular dystrophy (DMD) is currently tested in phase 3 clinical trials. The aim of this approach is to modulate splicing by skipping a specific exon to reframe disrupted dystrophin transcripts, allowing the synthesis of a partly functional dystrophin pr...

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Bibliographic Details
Main Authors: Christa L Tanganyika-de Winter, Hans Heemskerk, Tatyana G Karnaoukh, Maaike van Putten, Sjef J de Kimpe, Judith van Deutekom, Annemieke Aartsma-Rus
Format: Article
Language:English
Published: Elsevier 2012-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
antisense oligonucleotides
Duchenne muscular dystrophy
dystrophin
exon skipping
mouse models
therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253116301020

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