Self-complementary AAV Vectors; Advances and Applications

Ferrari, 1996, Second-strand synthesis is a rate limiting step for efficient transduction by recombinant adeno-associated virus vectors, J Virol, 70, 3227, 10.1128/JVI.70.5.3227-3234.1996 Fisher, 1996, Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis, J Virol, 70, 520, 10.1128/JVI.70.1.520-532.1996 Duan, 2000, Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus, J Clin Invest, 105, 1573, 10.1172/JCI8317 Sipo, 2007, Differential internalization and nuclear uncoating of self-complementary adeno-associated virus pseudotype vectors as determinants of cardiac cell transduction, Gene Ther, 14, 1319, 10.1038/sj.gt.3302987 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 Wang, 2007, Existence of transient functional double-stranded DNA intermediates during recombinant AAV transduction, Proc Natl Acad Sci USA, 104, 13104, 10.1073/pnas.0702778104 Nash, 2007, Purification of host cell enzymes involved in adeno-associated virus DNA replication, J Virol, 81, 5777, 10.1128/JVI.02651-06 Nash, 2008, Complete in vitro reconstitution of adeno-associated virus DNA replication requires the minichromosome maintenance complex proteins, J Virol, 82, 1458, 10.1128/JVI.01968-07 Nakai, 2000, Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo, J Virol, 74, 9451, 10.1128/JVI.74.20.9451-9463.2000 Berns, 1990, Parvovirus replication, Microbiol Rev, 54, 316, 10.1128/mr.54.3.316-329.1990 Duan, 2001, Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison, Mol Ther, 4, 383, 10.1006/mthe.2001.0456 Halbert, 2002, Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene, Nat Biotechnol, 20, 697, 10.1038/nbt0702-697 Zhong, 2008, Single-polarity recombinant adeno-associated virus 2 vector-mediated transgene expression in vitro and in vivo: mechanism of transduction, Mol Ther, 16, 290, 10.1038/sj.mt.6300376 Zhou, 2008, Adeno-associated virus of a single-polarity DNA genome is capable of transduction in vivo, Mol Ther, 16, 494, 10.1038/sj.mt.6300397 Alexander, 1994, DNA-damaging agents greatly increase the transduction of nondividing cells by adeno-associated virus vectors, J Virol, 68, 8282, 10.1128/JVI.68.12.8282-8287.1994 Alexander, 1996, Effects of gamma irradiation on the transduction of dividing and nondividing cells in brain and muscle of rats by adeno-associated virus vectors, Hum Gene Ther, 7, 841, 10.1089/hum.1996.7.7-841 Zhao, 2007, Adeno-associated virus 2-mediated gene transfer: role of a cellular serine/threonine protein phosphatase in augmenting transduction efficiency, Gene Ther, 14, 545, 10.1038/sj.gt.3302886 Zhong, 2004, Self-complementary adeno-associated virus 2 (AAV)-T cell protein tyrosine phosphatase vectors as helper viruses to improve transduction efficiency of conventional single-stranded AAV vectors in vitro and in vivo, Mol Ther, 10, 950, 10.1016/j.ymthe.2004.07.018 Kay, 2003, Looking into the safety of AAV vectors, Nature, 424, 251, 10.1038/424251b Miller, 2004, Adeno-associated virus vectors integrate at chromosome breakage sites, Nat Genet, 36, 767, 10.1038/ng1380 Russell, 2007, AAV vectors, insertional mutagenesis and cancer, Mol Ther, 15, 1740, 10.1038/sj.mt.6300299 Peng, 2000, Transduction of hepatocellular carcinoma (HCC) using recombinant adeno-associated virus (rAAV): in vitro and in vivo effects of genotoxic agents, J Hepatol, 32, 975, 10.1016/S0168-8278(00)80102-6 Carter, 1972, Adenovirus-associated virus multiplication. IX. Extent of transcription of the viral genome in vivo, J Virol, 10, 1118, 10.1128/JVI.10.6.1118-1125.1972 McCarty, 2001, Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis, Gene Ther, 8, 1248, 10.1038/sj.gt.3301514 Koeberl, 2008, AAV vector-mediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia, Mol Ther, 16, 665, 10.1038/mt.2008.15 Chapman, 1995, Single-stranded DNA-protein interactions in canine parvovirus, Structure, 3, 151, 10.1016/S0969-2126(01)00146-0 King, 2001, DNA helicase-mediated packaging of adeno-associated virus type 2 genomes into preformed capsids, EMBO J, 20, 3282, 10.1093/emboj/20.12.3282 Im, 1990, The AAV origin binding protein Rep68 is an ATP-dependent site-specific endonuclease with DNA helicase activity, Cell, 61, 447, 10.1016/0092-8674(90)90526-K McCarty, 2003, Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo, Gene Ther, 10, 2112, 10.1038/sj.gt.3302134 Wang, 2003, Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo, Gene Ther, 10, 2105, 10.1038/sj.gt.3302133 Wu, 2007, Self-complementary recombinant adeno-associated viral vectors: packaging capacity and the role of rep proteins in vector purity, Hum Gene Ther, 18, 171, 10.1089/hum.2006.088 Xu, 2005, Delivery of MDR1 small interfering RNA by self-complementary recombinant adeno-associated virus vector, Mol Ther, 11, 523, 10.1016/j.ymthe.2004.12.019 Wu, 2008, Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose, Mol Ther, 16, 280, 10.1038/sj.mt.6300355 Dong, 1996, Quantitative analysis of the packaging capacity of recombinant adeno-associated virus, Hum Gene Ther, 7, 2101, 10.1089/hum.1996.7.17-2101 Hermonat, 1997, The packaging capacity of adeno-associated virus (AAV) and the potential for wild-type-plus AAV gene therapy vectors, FEBS Lett, 407, 78, 10.1016/S0014-5793(97)00311-6 Allocca, 2008, Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice, J Clin Invest, 118, 1955, 10.1172/JCI34316 Grieger, 2005, Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry steps, J Virol, 79, 9933, 10.1128/JVI.79.15.9933-9944.2005 Miao, 2000, Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: cell cycling does not influence hepatocyte transduction, J Virol, 74, 3793, 10.1128/JVI.74.8.3793-3803.2000 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 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, 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, 2006, 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 liver, Blood, 107, 2653, 10.1182/blood-2005-10-4035 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 Koeberl, 2006, Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia, Gene Ther, 13, 1281, 10.1038/sj.gt.3302774 Hacker, 2005, Adeno-associated virus serotypes 1 to 5 mediated tumor cell directed gene transfer and improvement of transduction efficiency, J Gene Med, 7, 1429, 10.1002/jgm.782 Ren, 2005, Genomic stability of self-complementary adeno-associated virus 2 during early stages of transduction in mouse muscle in vivo, Hum Gene Ther, 16, 1047, 10.1089/hum.2005.16.1047 Andino, 2007, Rapid, widespread transduction of the murine myocardium using self-complementary Adeno-associated virus, Genet Vaccines Ther, 5, 13, 10.1186/1479-0556-5-13 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 McCown, 2005, Adeno-associated virus (AAV) vectors in the CNS, Curr Gene Ther, 5, 333, 10.2174/1566523054064995 Fu, 2003, Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain, Mol Ther, 8, 911, 10.1016/j.ymthe.2003.08.021 Fu, 2007, Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice, Gene Ther, 14, 1065, 10.1038/sj.gt.3302961 Foust, 2008, Neonatal intraperitoneal or intravenous injections of recombinant adeno-associated virus type 8 transduce dorsal root Ganglia and lower motor neurons, Hum Gene Ther, 19, 61, 10.1089/hum.2007.093 Kaspar, 2003, Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model, Science, 301, 839, 10.1126/science.1086137 Boulis, 2003, Intraneural colchicine inhibition of adenoviral and adeno-associated viral vector remote spinal cord gene delivery, Neurosurgery, 52, 381, 10.1227/01.NEU.0000044459.24519.3E Hollis, 2008, Efficient retrograde neuronal transduction utilizing self-complementary AAV1, Mol Ther, 16, 296, 10.1038/sj.mt.6300367 Alexander, 2008, Adeno-associated viral vectors and the retina, Adv Exp Med Biol, 613, 121, 10.1007/978-0-387-74904-4_13 Buch, 2008, AAV-mediated gene therapy for retinal disorders: from mouse to man, Gene Ther, 15, 849, 10.1038/gt.2008.66 Surace, 2008, Versatility of AAV vectors for retinal gene transfer, Vision Res, 48, 353, 10.1016/j.visres.2007.07.027 Bainbridge, 2008, Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis, N Engl J Med, 358, 2231, 10.1056/NEJMoa0802268 Maguire, 2008, Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis, N Engl J Med, 358, 2240, 10.1056/NEJMoa0802315 Yang, 2002, Virus-mediated transduction of murine retina with adeno-associated virus: effects of viral capsid and genome size, J Virol, 76, 7651, 10.1128/JVI.76.15.7651-7660.2002 Yokoi, 2007, Ocular gene transfer with self-complementary AAV vectors, Invest Ophthalmol Vis Sci, 48, 3324, 10.1167/iovs.06-1306 Natkunarajah, 2008, Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8, Gene Ther, 15, 463, 10.1038/sj.gt.3303074 Borras, 2006, Mechanisms of AAV transduction in glaucoma-associated human trabecular meshwork cells, J Gene Med, 8, 589, 10.1002/jgm.886 Shi, 2003, RGD inclusion in VP3 provides adeno-associated virus type 2 (AAV2)-based vectors with a heparan sulfate-independent cell entry mechanism, Mol Ther, 7, 515, 10.1016/S1525-0016(03)00042-X Stilwell, 2004, Role of viral vectors and virion shells in cellular gene expression, Mol Ther, 9, 337, 10.1016/j.ymthe.2003.11.007 Xiao, 2002, Adenovirus-facilitated nuclear translocation of adeno-associated virus type 2, J Virol, 76, 11505, 10.1128/JVI.76.22.11505-11517.2002 Komaromy, 2008, Targeting gene expression to cones with human cone opsin promoters in recombinant AAV, Gene Ther, 15, 1049, 10.1038/gt.2008.32 Chatterjee, 1999, Transduction of primitive human marrow and cord blood-derived hematopoietic progenitor cells with adeno-associated virus vectors, Blood, 93, 1882, 10.1182/blood.V93.6.1882.406k03_1882_1894 Fisher-Adams, 1996, Integration of adeno-associated virus vectors in CD34+ human hematopoietic progenitor cells after transduction, Blood, 88, 492, 10.1182/blood.V88.2.492.bloodjournal882492 Ponnazhagan, 1997, Adeno-associated virus type 2-mediated transduction of murine hematopoietic cells with long-term repopulating ability and sustained expression of a human globin gene in vivo, J Virol, 71, 3098, 10.1128/JVI.71.4.3098-3104.1997 Ponnazhagan, 1997, Adeno-associated virus type 2-mediated transduction in primary human bone marrow-derived CD34+ hematopoietic progenitor cells: donor variation and correlation of transgene expression with cellular differentiation, J Virol, 71, 8262, 10.1128/JVI.71.11.8262-8267.1997 Srivastava, 2005, Hematopoietic stem cell transduction by recombinant adeno-associated virus vectors: problems and solutions, Hum Gene Ther, 16, 792, 10.1089/hum.2005.16.792 Zhong, 2006, Evaluation of primitive murine hematopoietic stem and progenitor cell transduction in vitro and in vivo by recombinant adeno-associated virus vector serotypes 1 through 5, Hum Gene Ther, 17, 321, 10.1089/hum.2006.17.321 Han, 2008, Stable integration of recombinant adeno-associated virus vector genomes after transduction of murine hematopoietic stem cells, Hum Gene Ther, 19, 267, 10.1089/hum.2007.161 Maina, 2008, Recombinant self-complementary adeno-associated virus serotype vector-mediated hematopoietic stem cell transduction and lineage-restricted, long-term transgene expression in a murine serial bone marrow transplantation model, Hum Gene Ther, 19, 376, 10.1089/hum.2007.143 McCarty, 2004, Integration of adeno-associated virus (AAV) and recombinant AAV vectors, Annu Rev Genet, 38, 819, 10.1146/annurev.genet.37.110801.143717 Nakai, 2003, AAV serotype 2 vectors preferentially integrate into active genes in mice, Nat Genet, 34, 297, 10.1038/ng1179 Russell, 2003, AAV loves an active genome, Nat Genet, 34, 241, 10.1038/ng0703-241 Aldrich, 2006, Enhanced transduction of mouse bone marrow-derived dendritic cells by repetitive infection with self-complementary adeno-associated virus 6 combined with immunostimulatory ligands, Gene Ther, 13, 29, 10.1038/sj.gt.3302601 Veron, 2007, Major subsets of human dendritic cells are efficiently transduced by self-complementary adeno-associated virus vectors 1 and 2, J Virol, 81, 5385, 10.1128/JVI.02516-06 Lee, 2007, Efficient gene expression by self-complementary adeno-associated virus serotype 2 and 5 in various human cancer cells, Oncol Rep, 18, 611 Wu, 2007, Adeno-associated virus mediated gene transfer into lung cancer cells promoting CD40 ligand-based immunotherapy, Virology, 368, 309, 10.1016/j.virol.2007.07.006 Ding, 2003, Second-strand genome conversion of adeno-associated virus type 2 (AAV-2) and AAV-5 is not rate limiting following apical infection of polarized human airway epithelia, J Virol, 77, 7361, 10.1128/JVI.77.13.7361-7366.2003 Theiss, 2003, Enhancement of gene transfer with recombinant adeno-associated virus (rAAV) vectors into primary B-cell chronic lymphocytic leukemia cells by CpG-oligodeoxynucleotides, Exp Hematol, 31, 1223, 10.1016/j.exphem.2003.09.010 Choi, 2006, Host cell DNA repair pathways in adeno-associated viral genome processing, J Virol, 80, 10346, 10.1128/JVI.00841-06 Choi, 2005, Effects of adeno-associated virus DNA hairpin structure on recombination, J Virol, 79, 6801, 10.1128/JVI.79.11.6801-6807.2005