Drug discovery for Duchenne muscular dystrophy via utrophin promoter activation screening.

ABSTRACT:

Background

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.

Methodology/principal findings

We developed and validated a

SUBMITTER: Moorwood C 

PROVIDER: S-EPMC3197614 | biostudies-literature | 2011

REPOSITORIES: biostudies-literature