Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: alpha-, beta-, gamma-, or delta-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors.
Stedman
H
H
University of Pennsylvania Health System, Institute for Human Gene Therapy, Philadelphia 19104, USA.
Wilson
J M
JM
Finke
R
R
Kleckner
A L
AL
Mendell
J
J
D000256:Adenoviridae / Q000235:genetics; D000818:Animals; D002985:Clinical Protocols; D017321:Clinical Trials, Phase I as Topic; D003598:Cytoskeletal Proteins / Q000235:genetics* / Q000494:pharmacology; D049030:Dystroglycans; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000008:administration & dosage* / Q000235:genetics; D006801:Humans; D007273:Injections, Intramuscular; D008562:Membrane Glycoproteins / Q000235:genetics* / Q000494:pharmacology; D051379:Mice; D018345:Mice, Knockout; D009136:Muscular Dystrophies / Q000235:genetics / Q000628:therapy*; D049031:Sarcoglycans
D003598:Cytoskeletal Proteins; C483081:DAG1 protein, human; D008562:Membrane Glycoproteins; D049031:Sarcoglycans; D049030:Dystroglycans