title: Production of clinical-grade recombinant adeno-associated virus vectors.
abstract: Recombinant adeno-associated viral (rAAV) vectors are being evaluated in animal models and humans. Pre-clinical data demonstrating vector safety, efficiency and efficacy have been used to initiate human clinical trials. The clinical manufacture of rAAV vectors has supported phase I and phase II trials, showing that adeno-associated virus serotype 2 vectors are safe when administered to humans.
authors.affiliation: Powell Gene Therapy Center, University of Florida Genetics Institute, Department of Pediatrics, 1600 SW Archer Road, Gainesville, FL 32610-0266, USA.
authors.forename: Richard O
authors.initials: RO
authors.lastname: Snyder
authors.forename: Terence R
authors.initials: TR
authors.lastname: Flotte
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D003550:Cystic Fibrosis / Q000628:therapy; D004274:DNA, Recombinant / Q000235:genetics / Q000627:therapeutic use*; D000229:Dependovirus / Q000235:genetics* / Q000302:isolation & purification; D015967:Gene Expression Regulation, Viral; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics* / Q000302:isolation & purification / Q000627:therapeutic use*; D016679:Genome, Viral; D006801:Humans; D011995:Recombination, Genetic
chemical_list: D004274:DNA, Recombinant