Systematic Evaluation of AAV Vectors for Liver directed Gene Transfer in Murine Models

Nguyen, 2009, Ex vivo liver-directed gene therapy for the treatment of metabolic diseases: advances in hepatocyte transplantation and retroviral vectors, Curr Gene Ther, 9, 136, 10.2174/156652309787909481 Raper, 2002, A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency, Hum Gene Ther, 13, 163, 10.1089/10430340152712719 Manno, 2006, Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response, Nat Med, 12, 342, 10.1038/nm1358 Snyder, 1997, Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors, Nat Genet, 16, 270, 10.1038/ng0797-270 Scallan, 2006, Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice, Blood, 107, 1810, 10.1182/blood-2005-08-3229 Calcedo, 2009, Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses, J Infect Dis, 199, 381, 10.1086/595830 Mingozzi, 2007, CD8(+) T-cell responses to adeno-associated virus capsid in humans, Nat Med, 13, 419, 10.1038/nm1549 Gao, 2002, Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy, Proc Natl Acad Sci USA, 99, 11854, 10.1073/pnas.182412299 Gao, 2004, Clades of adeno-associated viruses are widely disseminated in human tissues, J Virol, 78, 6381, 10.1128/JVI.78.12.6381-6388.2004 Vandenberghe, 2009, Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints, Gene Ther, 10.1038/gt.2009.101 Sarkar, 2004, Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype, Blood, 103, 1253, 10.1182/blood-2003-08-2954 Davidoff, 2005, 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 models, Mol Ther, 11, 875, 10.1016/j.ymthe.2004.12.022 Sun, 2005, Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II, Mol Ther, 11, 57, 10.1016/j.ymthe.2004.10.004 Ziegler, 2007, Correction of the biochemical and functional deficits in fabry mice following AAV8-mediated hepatic expression of alpha-galactosidase A, Mol Ther, 15, 492, 10.1038/sj.mt.6300066 Wang, 2007, Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets, Hum Gene Ther, 18, 185, 10.1089/hum.2007.001 Gao, 2006, High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes, J Virol, 80, 6192, 10.1128/JVI.00526-06 Nathwani, 2007, Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates, Blood, 109, 1414, 10.1182/blood-2006-03-010181 Gao, 2009, Adeno-associated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specific T cell responses, Hum Gene Ther, 20, 930, 10.1089/hum.2009.060 Vandenberghe, 2006, Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid, Nat Med, 12, 967, 10.1038/nm1445 Lin, 2007, Vaccines based on novel adeno-associated virus vectors elicit aberrant CD8+ T-cell responses in mice, J Virol, 81, 11840, 10.1128/JVI.01253-07 Gao, 2006, Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates, Mol Ther, 13, 77, 10.1016/j.ymthe.2005.08.017 Limberis, 2009, Identification of the murine firefly luciferase-specific CD8 T-cell epitopes, Gene Ther, 16, 441, 10.1038/gt.2008.177 Wang, 2005, Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy, Blood, 105, 3079, 10.1182/blood-2004-10-3867 Bell, 2006, Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver, Mol Ther, 14, 34, 10.1016/j.ymthe.2006.03.008 Mood, 1963 Royston, 1993, Centile estimation command, Stata Tech Bull, 2, 12 Conover, 1999 Newson, 2001, Robust confidence intervals for the median and other percentile differences between two groups, Stata Tech Bull, 10, 30