Adeno-associated virus-mediated gene therapy for metabolic myopathy

Human Gene Therapy

Volumn 24, Issue 11, 2013, Pages 928-936

  Mah, Cathryn S ^a   Soustek, Meghan S ^a   Todd, A Gary ^a   McCall, Angela ^a   Smith, Barbara K ^a   Corti, Manuela ^a   Falk, Darin J ^a   Byrne, Barry J ^a  

^a UNIVERSITY OF FLORIDA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

GLYCOGEN; GLYCOGEN PHOSPHORYLASE; LENTIVIRUS VECTOR; LIPID; MITOCHONDRIAL DNA; PARVOVIRUS VECTOR; PLASMID DNA; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; RETROVIRUS VECTOR; SHORT HAIRPIN RNA;

ADENO ASSOCIATED VIRUS; ADENO ASSOCIATED VIRUS 1; ADENO ASSOCIATED VIRUS 2; ADENO ASSOCIATED VIRUS 5; ADENO ASSOCIATED VIRUS 8; ADENO ASSOCIATED VIRUS 9; DISEASE SEVERITY; DRUG INDICATION; EXERCISE TOLERANCE; GENE MUTATION; GLYCOGEN STORAGE DISEASE TYPE 2; GLYCOGEN STORAGE DISEASE TYPE 5; GLYCOGEN STORAGE DISEASE TYPE 7; HUMAN; METABOLIC MYOPATHY; MITOCHONDRIAL MYOPATHY; MUSCLE ATROPHY; MYOPATHY; NONHUMAN; PERSONALIZED MEDICINE; REVIEW; VERY LONG CHAIN ACYL COENZYME A DEHYDROGENASE DEFICIENCY; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; X LINKED MYOTUBULAR MYOPATHY;

ANIMALS; DEPENDOVIRUS; GENETIC THERAPY; GENETIC VECTORS; GLYCOGEN STORAGE DISEASE; HUMANS; LIPID METABOLISM DISORDERS; MITOCHONDRIAL MYOPATHIES;

EID: 84886793492     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2013.2514     Document Type: Review

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