Overview on DMD exon skipping
A Aartsma-Rus - Exon Skipping: Methods and Protocols, 2012 - Springer
Exon Skipping: Methods and Protocols, 2012•Springer
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.
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.
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.
Springer