| ［1］ 许瑞安, 陈凌,肖卫东. 分子基因药物学. 北京：北京大学出版社&北京大学医学出版社, 2009, 240. Xu R A, Chen L, Xiao W. Molecular Gene Medicine. Beijing:Peking University Press and Peking University Medical Press, 2009, 240.
［2］ Xu R A, Sun X, Tse L Y, et al. Longterm expression of angiostatin and suppression of liver metastatic cancer. Hepatology, 2003, 37(6):14511461.
［3］ Xu R A, Harrison P M, Chen M, et al. Cytoglobin protects against damageinduced liver fibrosis. Mol Ther, 2006, 13(6):10931100.
［4］ During M J, Xu R A, Young D, et al. Peroral gene therapy of lactose in tolerance using an adenoassociated virus vector. Nat Med, 1998, 4: 11311136.
［5］ During M J, Symes C W, Lawlor P A, et al. An oral vaccine against NMDAR1 with efficacy in experimental stroke and epilepsy. Science, 2000, 287(5457): 14531460.
［6］ Maguire A M, Simonelli F, Pierce E A, et al. Safety and efficacy of gene transfer for Leber’s congenital amaurosis.N Engl J Med, 2008, 358(21):22402248.
［7］ Bainbridge J W, Smith A J, Barker S S, et al. Effect of gene therapy on visual function in Leber’s congenital amaurosis. N Engl J Med, 2008, 358(21):22312239.
［8］ Hauswirth W W, Aleman T S, Kaushal S, et al. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adenoassociated virus gene vector: shortterm results of a phase I trial. Hum Gene Ther, 2008, 19(10): 979990.
［9］ Mueller C, Flotte T R.Clinical gene therapy using recombinant adenoassociated virus vectors. Gene Ther, 2008, 15, 858863.
［10］ Jacobson S G, Acland G M, Aguirre G D, et al. Safety of recombinant adenoassociated virus type 2RPE65 vector delivered by ocular subretinal injection. Mol Ther, 2006, 13:10741084.
［11］ Le Meur G, Stieger K, Smith A J, et al. Restoration of vision in RPE65de?cient Briard dogs using an AAV serotype 4 vector that speci?cally targets the retinal pigmented epithelium. Gene Ther,2007,14:292303.
［12］ Roman A J, Boye S L, Aleman T S, et al. Electroretinographic analyses of Rpe65mutant rd12 mice: developing an in vivo bioassay for human gene therapy trials of Leber congenital amaurosis. Mol Vis, 2007, 13:17011710.
［13］ Cideciyan A V, Aleman T S, Boye S L, et al. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci USA, 2008, 105:1511215117.
［14］ Manno C S, Chew A J, Hutchison S, et al. AAVmediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood, 2003, 101: 29632972.
［15］ Manno C S, Pierce G F, Arruda V R, et al. Successful transduction of liver in hemophilia by AAVFactor IX and limitations imposed by the host immune response. Nat Med, 2006, 12: 342347.
［16］ Zaiss A K, Muruve D A. Immunity to adenoassociated virus vectors in animals and humans: a continued challenge. Gene Ther, 2008, 15:808816.
［17］ Xiao W, Narendra C, Truneh A, et al. Humoral immunity to adenoassociated virus type 2 vectors following administration to murine and nonhuman primate muscle. J Virol, 2000, 74:24202425.
［18］ Xiao W, Chirmule N, Schnell M A, et al. Route of administration determines induction of Tcellindependent humoral responses to adenoassociated virus vectors. Mol Ther, 2000,1(4): 323329.
［19］ Daya S, Berns K I. Gene therapy using adenoassociated virus vectors. Clin Microbiol Rev, 2008, 21(4):583593.
［20］ Zabner J, Seiler M, Walters R, et al. Adenoassociated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer. J Virol, 2000, 74(8): 38523858.
［21］ Gao G, Vandenberghe L H, Alvira M R, et al. Clades of adenoassociated viruses are widely disseminated in human tissues. J Virol, 2004, 78(12):63816388.
［22］ Halbert C L, Allen J M, Miller A D. Adenoassociated virus type 6 (AAV6) vectors mediate efficient transduction of airway epithelial cells in mouse lungs compared to that of AAV2 vectors. J Virol, 2001, 75(14):66156624.
［23］ Limberis M P, Wilson J M. Adenoassociated virus serotype 9 vectors transduce murine alveolar and nasal epithelia and can be readministered. Proc Natl Acad Sci USA, 2006, 103(35):1299312998.
［24］ Mori S, Takeuchi T, Enomoto Y, et al. Tissue distribution of cynomolgus adenoassociated viruses AAV10, AAV11, and AAVcy.7 in naturally infected monkeys. Arch Virol, 2008, 153(2): 375380.
［25］ Schmidt M, Voutetakis A, Afione S, et al. Adenoassociated virus type 12 (AAV12): a novel AAV serotype with sialic acid and heparan sulfate proteoglycanindependent transduction activity. J. Virol, 2008, 82:13991406.
［26］ Xu R A, Janson C G, Mastakov M, et al. Quantitative comparison of expression with adenoassociated virus (AAV2) brainspecific gene cassettes. Gene Ther, 2001, 8:13231332.
［27］ Wang Q Z, Lv Y H, Xu R A. Dosereponse and control of adenoassociated viral vectors based prechilical and clinical gene therapy. Chinese Journal Clinical Pharmacology and Therapeutics, 2008, 13(10):11821194.
［28］ Janson C, McPhee S, Bilaniuk L, et al. Clinical protocol. Gene therapy of Canavan disease: AAV2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain. Hum Gene Ther, 2002, 13:13911412v
［29］ Lu H, Qu G, Xu R, et al. Systemic elimination of de novo capsid protein synthesis from replication competent AAV contamination. Mol Ther, 2009, (in press).
［30］ Kaplitt M G, Feigin A, Tang C, et al. Safety and tolerability of gene therapy with an adenoassociated virus (AAV) borne GAD gene for Parkinson’s disease: an open label, phase I trial. Lancet, 2007, 23; 369(9579):20972105.
［31］ Apparailly F, Khoury M, Vervoordeldonk M J, et al. Adenoassociated virus pseudotype 5 vector improves gene transfer in arthritic joints. Hum Gene Ther, 2005, 16(4):426434.
［32］ Cao L, Lin E J D, Cahill M C, et al. Molecular therapy of obesity and diabetes by a physiological autoregulatory approach. Nat Med, 2009, 15:447454.
［33］吕颖慧, 王启钊，肖卫东等.自身互补型腺相关病毒载体研究进展.生物工程学报, 2009, 25(5): 658664. Lv Y, Wang Q, Xiao W, et al. Chin J Biotech,2009, 25(5): 658664.
［34］ Liu Y L, Wagner K, Robinson N, et al. Optimized production of hightiter recombinant adenoassociated virus in roller bottles. Biotechniques, 2003, 34:184189.
［35］ 刁勇, 许瑞安. 细胞生物技术实验指南.北京：化学化工出版社, 2009: 306337. Diao Y, Xu R A.Protocols of Cellular and Molecular Biotechnology. Beijing:Peking Chemical Industry Press, 2009: 306337.
［36］ Chang J, Nicolas E, Marks D, et al. miR122, a mammalian liverspecific microRNA, is processed from hcr mRNA and may downregulate the high affinity cationic amino acid transporter CAT1. RNA Biol, 2004, 1(2):106113.
［37］ Wang J, Xie J, Lu H, et al. Existence of transient functional doublestranded DNA intermediates during recombinant AAV transduction. Proc Natl Acad Sci USA, 2007, 104(32): 1310413109.
［38］ Nathwani A C, Gray J T, McIntosh J, et al. Safe and efficient transduction of the liver after peripheral vein infusion of selfcomplementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates. Blood, 2007, 109(4): 1414 1421.
［39］ Nathwani A C, Gray J T, McIntosh J, et al. Selfcomplementary adenoassociated virus vectors containing a novel liverspecific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver. Blood, 2006, 107(7): 26532661.
［40］ Miller D G, Trobridge G D, Petek L M, et al. Largescale analysis of adenoassociated virus vector integration sites in normal human cells. J Virol, 2005, 79: 1143411442.
［41］ Nakai H, Wu X, Fuess S, et al. Largescale molecular characterization of adenoassociated virus vector integration in mouse liver. J Virol, 2005, 79: 36063614.
［42］ Smith R H. Adenoassociated virus integration: virus versus vector. Gene Ther, 2008, 15:817822.
［43］ Wang Q Z, Lv Y H, Yong D, et al. The design of vectors for RNAi delivery system. Curr Pharm Design, 2008, 14(13):13271340.
［44］ Shu J B, Diao Y, Xiao W, et al. The development trend of chimeric vector of adenovirus/adeno associated viral. J Huaqiao University (Natural Science), 2008, 29(2):172176.
［45］ Corte′s M L, Oehmig A, Saydam O, et al. Targeted integration of functional human ATM cDNA into genome mediated by HSV/AAV hybrid amplicon vector. Mol Ther, 2008, 16: 8188. |
