Molecular pathophysiology and targeted therapeutics for muscular dystrophy

Experimental therapeutics of the muscular dystrophies has made impressive advances on several fronts. Adeno-associated virus has emerged as the clear ‘vector of choice’ for muscle gene delivery, with successful functional rescue of dystrophic muscle in rodent models. Correction of the dystrophin gene mutation in a dog model has been reported, and several reports of progress on myogenic stem cell characterization are resurrecting cell transplantation as a possible therapeutic approach. The downstream consequences of dystrophin deficiency are being defined quickly using microarray experiments, and drugs targeting specific biochemical pathways are being tested rapidly in animal models. Such targeted drug discoveries, which are discussed in this article, have begun to be implemented in human clinical trials.