title: Manufacturing and regulatory strategies for clinical AAV2-hRPE65.
abstract: Recombinant adeno-associated virus (AAV) -based vectors expressing therapeutic gene products have shown great promise for human gene therapy. A recent milestone has been the safety and efficacy observed using recombinant AAV2 expressing retinal pigment epithelial associated 65KDa protein for Leber Congenital Amaurosis. This review summarizes manufacturing and characterization of 'AAV2-hRPE65v2', the vector used in one completed Phase I/II clinical trial. Regulatory challenges and strategies that were successfully used for this groundbreaking trial are described.
authors: Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia PA, USA.
authors: J Fraser
authors: JF
authors: Wright
authors: Jennifer
authors: J
authors: Wellman
authors: Katherine A
authors: KA
authors: High
mesh_terms: D002352:Carrier Proteins / Q000235:genetics*; D002986:Clinical Trials as Topic; D000229:Dependovirus; D005136:Eye Proteins / Q000235:genetics*; D018014:Gene Transfer Techniques / Q000592:standards*; D015316:Genetic Therapy / Q000331:legislation & jurisprudence / Q000592:standards; D005822:Genetic Vectors / Q000096:biosynthesis; D006801:Humans; D057130:Leber Congenital Amaurosis / Q000628:therapy*; D019745:cis-trans-Isomerases; D000099178:Retinoid Isomerohydrolase
chemical_list: D002352:Carrier Proteins; D005136:Eye Proteins; D019745:cis-trans-Isomerases; D000099178:Retinoid Isomerohydrolase