title: Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with Pompe disease.
authors.affiliation: Departments of Pediatrics and Surgery, University of Florida , Gainesville, FL 32611.
authors.forename: Principal Investigators Barry J
authors.initials: PI
authors.lastname: Byrne
authors.forename: Shelley
authors.initials: S
authors.lastname: Collins
authors.forename: Coinvestigators Cathryn
authors.initials: CC
authors.lastname: Mah
authors.forename: Barbara
authors.initials: B
authors.lastname: Smith
authors.forename: Thomas
authors.initials: T
authors.lastname: Conlon
authors.forename: Subinvestigators Danny
authors.initials: SD
authors.lastname: Martin
authors.forename: Manuela
authors.initials: M
authors.lastname: Corti
authors.forename: Brian
authors.initials: B
authors.lastname: Cleaver
authors.forename: Saleem
authors.initials: S
authors.lastname: Islam
authors.forename: Lee Ann
authors.initials: LA
authors.lastname: Lawson
mesh_terms: D000293:Adolescent; D000818:Animals; D002648:Child; D002675:Child, Preschool; D000229:Dependovirus / Q000235:genetics*; D003964:Diaphragm; D004353:Drug Evaluation, Preclinical; D005260:Female; D005500:Follow-Up Studies; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics*; D006009:Glycogen Storage Disease Type II / Q000628:therapy*; D006801:Humans; D008279:Magnetic Resonance Imaging; D008297:Male; D051379:Mice; D011817:Rabbits; D015898:Tomography Scanners, X-Ray Computed; D000520:alpha-Glucosidases / Q000235:genetics* / Q000378:metabolism
chemical_list: D000520:alpha-Glucosidases