Article
Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues.
Nucleic acid therapeutics - 1 Oct 2017
Aartsma-Rus Annemieke, Straub Volker, Hemmings Robert, Haas Manuel, Schlosser-Weber Gabriele, Stoyanova-Beninska Violeta, Mercuri Eugenio, Muntoni Francesco, Sepodes Bruno, Vroom Elizabeth, Balabanov Pavel
Abstract excerpt
Duchenne muscular dystrophy (DMD) is a rare, severe, progressive muscle-wasting disease leading to disability and premature death. Patients lack the muscle membrane-stabilizing protein dystrophin. Antisense oligonucleotide (AON)-mediated exon skipping is a therapeutic approach that aims to induce production of partially functional dystrophins. Recently, an AON targeting exon 51 became the first of its class to be...
Topics
- Animals
- Disease Models, Animal
- Drug Approval
- Dystrophin
- Exons
- Genetic Therapy
- Humans
- Mice
- Muscular Dystrophy, Duchenne
- Mutation