title: 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.
authors.affiliation: University of Pennsylvania Health System, Institute for Human Gene Therapy, Philadelphia 19104, USA.
authors.forename: H
authors.initials: H
authors.lastname: Stedman
authors.forename: J M
authors.initials: JM
authors.lastname: Wilson
authors.forename: R
authors.initials: R
authors.lastname: Finke
authors.forename: A L
authors.initials: AL
authors.lastname: Kleckner
authors.forename: J
authors.initials: J
authors.lastname: Mendell
mesh_terms: 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
chemical_list: D003598:Cytoskeletal Proteins; C483081:DAG1 protein, human; D008562:Membrane Glycoproteins; D049031:Sarcoglycans; D049030:Dystroglycans