title: High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes.
abstract: Adeno-associated virus (AAV) vectors are being considered for in vivo applications of gene therapy in the treatment of a variety of disorders. This study evaluates the biology of second-generation vectors based on the novel serotypes AAV7 and AAV8 and containing self-complementary genomes in the nonhuman primate liver. Stable levels of transgene expression were achieved in cynomolgus macaques and suggest efficiencies at least 2 log higher than what could be achieved with AAV2 vectors using traditional single-stranded genomes. Analysis of DNAs from tissues revealed high levels of vector in the liver that appeared proportional to the relative amounts of transgene expression.
authors.affiliation: Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, Philadelphia, PA 19104-3403, USA.
authors.forename: Guang-Ping
authors.initials: GP
authors.lastname: Gao
authors.forename: You
authors.initials: Y
authors.lastname: Lu
authors.forename: Xun
authors.initials: X
authors.lastname: Sun
authors.forename: Julie
authors.initials: J
authors.lastname: Johnston
authors.forename: Roberto
authors.initials: R
authors.lastname: Calcedo
authors.forename: Rebecca
authors.initials: R
authors.lastname: Grant
authors.forename: James M
authors.initials: JM
authors.lastname: Wilson
mesh_terms: D000818:Animals; D018076:DNA, Complementary; D000229:Dependovirus / Q000235:genetics*; D015870:Gene Expression; D015316:Genetic Therapy; D005822:Genetic Vectors; D016679:Genome, Viral / Q000235:genetics*; D008099:Liver / Q000378:metabolism* / Q000821:virology*; D008252:Macaca fascicularis; D012703:Serotyping; D019076:Transgenes / Q000235:genetics*
chemical_list: D018076:DNA, Complementary