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Methods Mol Biol. 2012;867:97-116. doi: 10.1007/978-1-61779-767-5_7.

Overview on DMD exon skipping.

Author information

1
Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands. a.m.rus@lumc.nl

Abstract

Antisense-mediated exon skipping to restore the disrupted dystrophin reading frame is currently in clinical trials for Duchenne muscular dystrophy. This chapter describes the rationale of this approach and gives an overview of in vitro and in vivo experiments with antisense oligonucleotides and antisense genes. Finally, an overview of clinical trials is given and outstanding questions and hurdles are discussed.

PMID:
22454057
DOI:
10.1007/978-1-61779-767-5_7
[Indexed for MEDLINE]

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