Article
Overview on DMD exon skipping.
Methods in molecular biology (Clifton, N.J.) - 1 Jan 2012
Aartsma-Rus Annemieke
Abstract excerpt
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.
Topics
- Animals
- Dystrophin
- Exons
- Genetic Therapy
- Humans
- Muscular Dystrophy, Duchenne
- Mutation
- Oligonucleotides, Antisense
- Reading Frames