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Overview on DMD Exon Skipping

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Exon Skipping

Part of the book series: Methods in Molecular Biology ((MIMB,volume 867))

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.

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Acknowledgments

The author receives funding from ZonMw (the Netherlands), the Dutch Duchenne Parent Project (the Netherlands), Spieren voor spieren (Prinses Beatrix Foundation, the Netherlands), and the European Union (LUMC is partner in the TREAT-NMD network of excellence (LSHM-CT-2006-036825) and the BIO-NMD project (HEALTH-F2-2009-241665)). The LUMC participates in the Center for Biomedical Genetics (the Netherlands) and the Center for Medical Systems Biology (the Netherlands).

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Authors and Affiliations

  1. Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands

    Annemieke Aartsma-Rus

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  1. Annemieke Aartsma-Rus
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Correspondence to Annemieke Aartsma-Rus .

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Editors and Affiliations

  1. Dept. Human & Clinical Genetics, Leiden University Medical Center, Einthovenweg 20, Leiden, 2333 ZC, Netherlands

    Annemieke Aartsma-Rus

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Conflict of Interest

The author reports being employed by LUMC and coinventor of patent applications for antisense sequences and the exon skipping technology. LUMC has licensed the rights to the patents on PRO051 exclusively to Prosensa Therapeutics. The inventors specified on the patents (including the author) are jointly entitled to a share of any future royalties paid to LUMC, should the therapy eventually be brought to the market.

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Aartsma-Rus, A. (2012). Overview on DMD Exon Skipping. In: Aartsma-Rus, A. (eds) Exon Skipping. Methods in Molecular Biology, vol 867. Humana Press. https://doi.org/10.1007/978-1-61779-767-5_7

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  • DOI: https://doi.org/10.1007/978-1-61779-767-5_7

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