Molecular pathophysiology and targeted therapeutics for muscular dystrophy

Experimental therapeutics of the muscular dystrophies has made impressive a dvances on several fronts. Adeno-associated virus has emerged as the clear 'vector of choice' for muscle gene delivery, with successful functional res cue of dystrophic muscle in rodent models. Correction of the dystrophin gen e mutation in a dog model has been reported, and several reports of progres s on myogenic stem cell characterization are resurrecting cell transplantat ion as a possible therapeutic approach. The downstream consequences of dyst rophin deficiency are being defined quickly using microarray experiments, a nd drugs targeting specific biochemical pathways are being tested rapidly i n animal models. Such targeted drug discoveries, which are discussed in thi s article, have begun to be implemented in human clinical trials.