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- Title
Pharmacological strategies for muscular dystrophy.
- Authors
Khurana, Tejvir S.; Davies, Kay E.
- Abstract
Duchenne muscular dystrophy (DMD) is a fatal, genetic disorder whose relentless progression underscores the urgency for developing a cure. Although Duchenne initiated clinical trials roughly 150 years ago, therapies for DMD remain supportive rather than curative. A paradigm shift towards developing rational therapeutic strategies occurred with identification of the DMD gene. Gene- and cell-based therapies designed to replace the missing gene and/or dystrophin protein have achieved varying degrees of success. However, pharmacological strategies not designed to replace dystrophin per se appear promising, and can circumvent many hurdles hampering gene- and cell-based therapy. Here, we will review present pharmacological strategies, in particular those dealing with functional substitution of dystrophin by utrophin and enhancing muscle progenitor commitment by myostatin blockade, with a view toward facilitating drug discovery for DMD.
- Subjects
DUCHENNE muscular dystrophy; PHARMACOLOGY; DYSTROPHIN
- Publication
Nature Reviews Drug Discovery, 2003, Vol 2, Issue 5, p379
- ISSN
1474-1776
- Publication type
Article
- DOI
10.1038/nrd1085