Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

Molecular Therapy - Tập 16 Số 6 - Trang 1073-1080 - 2008
Carmela Zincarelli1, Stephen Soltys1,2, Giuseppe Rengo1,2, Joseph E. Rabinowitz1
1Center for Translational Medicine, Department of Medicine, Thomas Jefferson University, Philadelphia, Pennsylvania, USA
2The George Zallie and Family Laboratory for Cardiovascular Gene Therapy, Department of Medicine, Thomas Jefferson University, Philadelphia, Pennsylvania, USA

Tóm tắt

Từ khóa


Tài liệu tham khảo

Berns, 1995, The cryptic life style of adeno-associated virus, Bioassays, 17, 237, 10.1002/bies.950170310

Bartlett, 2000, Infectious entry pathway of adeno-associated virus and adeno-associated virus vectors, J Virol, 74, 2777, 10.1128/JVI.74.6.2777-2785.2000

Duan, 1999, Dynamin is required for recombinant adeno-associated virus type 2 infection, J Virol, 73, 10371, 10.1128/JVI.73.12.10371-10376.1999

Douar, 2001, Intracellular trafficking of adeno-associated virus vectors: routing to the late endosomal compartment and proteasome degradation, J Virol, 75, 1824, 10.1128/JVI.75.4.1824-1833.2001

Thomas, 2004, Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors, J Virol, 78, 3110, 10.1128/JVI.78.6.3110-3122.2004

Chiorini, 1997, Cloning of adeno-associated virus type 4 (AAV4) and generation of recombinant AAV4 particles, J Virol, 71, 6823, 10.1128/JVI.71.9.6823-6833.1997

Rutledge, 1998, Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2, J Virol, 72, 309, 10.1128/JVI.72.1.309-319.1998

Chiorini, 1999, Cloning and characterization of adeno-associated virus type 5, J Virol, 73, 1309, 10.1128/JVI.73.2.1309-1319.1999

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

Gao, 2003, Adeno-associated viruses undergo substantial evolution in primates during natural infections, Proc Natl Acad Sci USA, 100, 6081, 10.1073/pnas.0937739100

Rabinowitz, 2002, Cross-packaging of single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity, J Virol, 76, 791, 10.1128/JVI.76.2.791-801.2002

Burger, 2004, Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system, Mol Ther, 10, 302, 10.1016/j.ymthe.2004.05.024

Cearley, 2006, Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain, Mol Ther, 13, 528, 10.1016/j.ymthe.2005.11.015

Taymans, 2007, Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain, Hum Gene Ther, 18, 195, 10.1089/hum.2006.178

Sondhi, 2007, Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector, Mol Ther, 15, 481, 10.1038/sj.mt.6300049

Klein, 2008, AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter and purification method, Mol Ther, 16, 89, 10.1038/sj.mt.6300331

White, 2004, Targeted gene delivery to vascular tissue in vivo by tropism-modified adeno-associated virus vectors, Circulation, 109, 513, 10.1161/01.CIR.0000109697.68832.5D

Work, 2006, Vascular bed-targeted in vivo gene delivery using tropism-modified adeno-associated viruses, Mol Ther, 13, 683, 10.1016/j.ymthe.2005.11.013

Halbert, 2000, Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes, J Virol, 74, 1524, 10.1128/JVI.74.3.1524-1532.2000

Halbert, 2001, Adeno-associated virus type 6 (AAV6) vectors mediate efficient transduction of airway epithelial cells in mouse lungs compared to that of AAV2 vectors, J Virol, 75, 6615, 10.1128/JVI.75.14.6615-6624.2001

Grimm, 2006, Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype, J Virol, 80, 426, 10.1128/JVI.80.1.426-439.2006

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

Inagaki, 2006, Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8, Mol Ther, 14, 45, 10.1016/j.ymthe.2006.03.014

Nakai, 2005, Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver, J Virol, 79, 3606, 10.1128/JVI.79.6.3606-3614.2005

Chao, 2000, Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors, Mol Ther, 2, 619, 10.1006/mthe.2000.0219

Blankinship, 2004, Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6, Mol Ther, 10, 671, 10.1016/j.ymthe.2004.07.016

Wang, 2005, Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart, Nat Biotechnol, 23, 321, 10.1038/nbt1073

Li, 2003, Efficient and long-term intracardiac gene transfer in delta-sarcoglycan-deficienty hamster by adeno-associated virus-2 vectors, Gene Ther, 10, 1807, 10.1038/sj.gt.3302078

Gregorevic, 2004, Systemic delivery of genes to striated muscles using adeno-associated viral vectors, Nat Med, 10, 828, 10.1038/nm1085

Müller, 2006, Improved cardiac gene transfer by transcriptional and transductional targeting of adeno-associated viral vectors, Cardiovasc Res, 70, 70, 10.1016/j.cardiores.2005.12.017

Pacak, 2006, Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo, Circ Res, 99, e3, 10.1161/01.RES.0000237661.18885.f6

Palomeque, 2007, Efficiency of eight different AAV serotypes in transducing rat myocardium in vivo, Gene Ther, 14, 989, 10.1038/sj.gt.3302895

Wu, 2001, Noninvasive optical imaging of firefly luciferase reporter gene expression in skeletal muscles of living mice, Mol Ther, 4, 297, 10.1006/mthe.2001.0460

Lipshutz, 2001, In utero delivery of adeno-associated viral vectors: intraperitoneal gene transfer produces long-term expression, Mol Ther, 3, 284, 10.1006/mthe.2001.0267

Weissleder, 2003, Shedding light onto live molecular targets, Nat Med, 9, 123, 10.1038/nm0103-123

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

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

Grimm, 2003, Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy, Blood, 102, 2412, 10.1182/blood-2003-02-0495

Haberman, 1999, Production of recombinant adeno-associated viral vectors and use in vitro and in vivo administration, 4.17.1

Rabinowitz, 1996, Targeted mutation of Ncam to produce a secreted molecule results in a dominant embryonic lethality, Proc Natl Acad Sci USA, 93, 6421, 10.1073/pnas.93.13.6421