Molecular pathophysiology and targeted therapeutics for muscular dystrophy.

  title={Molecular pathophysiology and targeted therapeutics for muscular dystrophy.},
  author={Eric P. Hoffman and Devin Dressman},
  journal={Trends in pharmacological sciences},
  volume={22 9},
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… CONTINUE READING

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