High-Level Transgene Expression in Nonhuman Primate Liver with Novel Adeno-Associated Virus Serotypes Containing Self-Complementary Genomes
- 15 June 2006
- journal article
- research article
- Published by American Society for Microbiology in Journal of Virology
- Vol. 80 (12) , 6192-6194
- https://doi.org/10.1128/jvi.00526-06
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.Keywords
This publication has 10 references indexed in Scilit:
- Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liverBlood, 2006
- Biology of AAV Serotype Vectors in Liver-Directed Gene Transfer to Nonhuman PrimatesMolecular Therapy, 2006
- Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate modelsMolecular Therapy, 2005
- Clades of Adeno-Associated Viruses Are Widely Disseminated in Human TissuesJournal of Virology, 2004
- Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivoGene Therapy, 2003
- A Limited Number of Transducible Hepatocytes Restricts a Wide-Range Linear Vector Dose Response in Recombinant Adeno-Associated Virus-Mediated Liver TransductionJournal of Virology, 2002
- Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapyProceedings of the National Academy of Sciences, 2002
- Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesisGene Therapy, 2001
- Design and Packaging of Adeno-Associated Virus Gene Targeting VectorsJournal of Virology, 2000
- Nonrandom Transduction of Recombinant Adeno-Associated Virus Vectors in Mouse Hepatocytes In Vivo: Cell Cycling Does Not Influence Hepatocyte TransductionJournal of Virology, 2000