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.