Dmd – therapy

Duchenne muscular dystrophy (DMD) is a common and fatal neuromuscular disease caused by loss-of-function mutations in the DMD gene which encodes dystrophin. Such genetic defects are amenable to correction by genome editing. However, DMD can be caused by an extensive list of mutations which will require a large volume of personalized gene therapies to tackle. This demand cannot be met in a reasonable timeframe with current development pipelines. Producing a DMD gene therapy requires assessing the approach's ability to restore the dystrophin protein in vitro prior to in vivo studies.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research