Adeno-associated virus-mediated gene therapy for metabolic myopathy.

  title={Adeno-associated virus-mediated gene therapy for metabolic myopathy.},
  author={Cathryn S. Mah and Meghan S. Soustek and Adrian Gary Todd and Angela R McCall and Barbara Kellerman Smith and Manuela Corti and Darin J. Falk and Barry Byrne},
  journal={Human gene therapy},
  volume={24 11},
Metabolic myopathies are a diverse group of rare diseases in which impaired breakdown of stored energy leads to profound muscle dysfunction ranging from exercise intolerance to severe muscle wasting. Metabolic myopathies are largely caused by functional deficiency of a single gene and are generally subcategorized into three major types of metabolic disease: mitochondrial, lipid, or glycogen. Treatment varies greatly depending on the biochemical nature of the disease, and unfortunately no… CONTINUE READING