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- Title
Drug discovery for Duchenne muscular dystrophy via utrophin promoter activation screening.
- Authors
Moorwood, Catherine; Lozynska, Olga; Suri, Neha; Napper, Andrew D; Diamond, Scott L; Khurana, Tejvir S
- Abstract
Duchenne muscular dystrophy (DMD) is a devastating muscle wasting disease caused by mutations in dystrophin, a muscle cytoskeletal protein. Utrophin is a homologue of dystrophin that can functionally compensate for its absence when expressed at increased levels in the myofibre, as shown by studies in dystrophin-deficient mice. Utrophin upregulation is therefore a promising therapeutic approach for DMD. The use of a small, drug-like molecule to achieve utrophin upregulation offers obvious advantages in terms of delivery and bioavailability. Furthermore, much of the time and expense involved in the development of a new drug can be eliminated by screening molecules that are already approved for clinical use.
- Publication
PloS one, 2011, Vol 6, Issue 10, pe26169
- ISSN
1932-6203
- Publication type
Journal Article
- DOI
10.1371/journal.pone.0026169