Gene therapy: Promises and problems

Annual Review of Genomics and Human Genetics

Volumn 2, Issue , 2001, Pages 177-211

  Pfeifer, Alexander ^a   Verma, Inder M ^a  

^a SALK INSTITUTE FOR BIOLOGICAL STUDIES   (United States)

Author keywords

Gene therapy trials; Gene transfer; Viral vectors

Indexed keywords

ADENOVIRUS VECTOR; LENTIVIRUS VECTOR; VIRUS DNA; VIRUS RNA; VIRUS VECTOR;

ADENOVIRUS; CLINICAL FEATURE; DNA DETERMINATION; GENE DELIVERY SYSTEM; GENE THERAPY; GENE TRANSFER; GENETIC ANALYSIS; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS; LENTIVIRINAE; MURINE LEUKEMIA VIRUS; NONHUMAN; RETROVIRUS; REVIEW; RNA ANALYSIS; TARGET CELL; VIRAL GENE DELIVERY SYSTEM; VIRUS GENE; VIRUS TRANSFORMATION;

DNA VIRUSES; GENE THERAPY; GENETIC VECTORS; HUMANS; RNA VIRUSES;

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; DNA VIRUSES; HUMAN IMMUNODEFICIENCY VIRUS; LENTIVIRUS; MURINAE; MURINE LEUKEMIA VIRUS; RNA VIRUSES; UNIDENTIFIED RETROVIRUS;

EID: 0035778751     PISSN: 15278204     EISSN: None     Source Type: Book Series    
DOI: 10.1146/annurev.genom.2.1.177     Document Type: Review

References (190)

1. Abonour R, Williams DA, Einhorn L, Hall KM, Chen J, et al. 2000. Efficient retrovirus-mediated transfer of the multi-drug resistance 1 gene into autologous human long-term repopulating hematopoietic stem cells. Nat. Med. 6:652-58
2. Agha-Mohammadi S, Lotze MT 2000. Regulatable systems: Applications in gene therapy and replicating viruses. J. Clin. Invest. 105:1177-83
3. Albritton LM, Tseng L, Scadden D, Cunningham JM. 1989. A putative murine ecotropic retrovirus receptor gene encodes a multiple membrane-spanning protein and confers susceptibility to virus infection. Cell 57:659-66
4. Alemany R, Balague C, Curiel DT. 2000. Replicative adenoviruses for cancer therapy. Nat. Biotechnol. 18:723-27
5. Amalfitano A, Hauser MA, Hu H, Serra D, Begy CR, Chamberlain JS. 1998. Production and characterization of improved adenovirus vectors with the E1, E2b, and E3 genes deleted. J. Virol. 72:926-33
6. Armentano D, Smith MP, Sookdeo CC, Zabner J, Perricone MA, et al. 1999. E4ORF3 requirement for achieving long-term transgene expression from the cytomegalovirus promoter in adenovirus vectors. J. Virol. 73:7031-34
7. Armentano D, Yu SF, Kantoff PW, von Ruden T, Anderson WF, Gilboa E. 1987. Effect of internal viral sequences on the utility of retroviral vectors. J. Virol. 61:1647-50
8. Armentano D, Zabner J, Sacks C, Sookdeo CC, Smith MP, et al. 1997. Effect of the E4 region on the persistence of transgene expression from adenovirus vectors. J. Virol. 71:2408-16
9. Bender MA, Palmer TD, Gelinas RE, Miller AD. 1987. Evidence that the packaging signal of Moloney murine leukemia virus extends into the gag region. J. Virol. 61:1639-46
10. Bergelson JM, Cunningham JA, Droguett G, Kurt-Jones EA, Krithivas A, et al. 1997. Isolation of a common receptor for Coxsackie B viruses and adenoviruses 2 and 5. Science 275:1320-23
11. Berkner KL, Sharp PA. 1983. Generation of adenovirus by transfection of plasmids. Nucleic Acids Res. 11:6003-20
12. Berkowitz RD, Ilves H, Plavec I, Veres G. 2001. Gene transfer systems derived from visna virus: Analysis of virus production and infectivity. Virology 279:116-29
13. Berns KI. 1996. Parvoviridae: The Viruses and Their Replication. See Ref. 41a, pp. 2173-97
14. Berns KI, Linden RM. 1995. The cryptic lifestyle of adeno-associated virus. BioEssays 17:237-45
15. Bett AJ, Haddara W, Prevec L, Graham FL. 1994. An efficient and flexible system for construction of adenovirus vectors with insertions or deletions in early regions 1 and 3. Proc. Natl. Acad. Sci. USA 91:8802-6
16. Bett AJ, Prevec L, Graham FL. 1993. Packaging capacity and stability of human adenovirus type 5 vectors. J. Virol. 67:5911-21
17. Bischoff JR, Kirn DH, Williams A, Heise C, Horn S, et al. 1996. An adenovirus mutant that replicates selectively in p53-deficient human tumor cells. Science 274:373-76
18. Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, et al. 1995. T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years. Science 270:475-80
19. Brown PO. 1997. Integration. See Ref. 30a pp. 161-203
20. Bukrinsky MI, Haggerty S, Dempsey MP, Sharova N, Adzhubel A, et al. 1993. A nuclear localization signal within HIV-1 matrix protein that governs infection of nondividing cells. Nature 365:666-69
21. Burns JC, Friedmann T, Driever W, Burrascano M, Yee JK. 1993. Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: Concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells. Proc. Natl. Acad. Sci. USA 90:8033-37
22. (-) human hematopoietic cells by HIV-1-based lentiviral vectors. Proc. Natl. Acad. Sci. USA 96:2988-93
23. Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E, et al. 2000. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288:669-72
24. Chambers R, Gillespie GY, Soroceanu L, Andreansky S, Chatterjee S, et al. 1995. Comparison of genetically engineered herpes simplex viruses for the treatment of brain tumors in a scid mouse model of human malignant glioma. Proc. Natl. Acad. Sci. USA 92:1411-15
25. Chartier C, Degryse E, Gantzer M, Dieterle A, Pavirani A, Mehtali M. 1996. Efficient generation of recombinant adenovirus vectors by homologous recombination in Escherichia coli. J. Virol. 70:4805-10
26. Chen ST, Iida A, Guo L, Friedmann T, Yee JK. 1996. Generation of packaging cell lines for pseudotyped retroviral vectors of the G protein of vesicular stomatitis virus by using a modified tetracycline inducible system. Proc. Natl. Acad. Sci. USA 93:10057-62
27. Chirmule N, Propert K, Magosin S, Qian Y, Qian R, Wilson J. 1999. Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther. 6:1574-83
28. Clark KR, Liu X, McGrath JP, Johnson PR. 1999. Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses. Hum. Gene Ther. 10:1031-39
29. Coffin JM. 1992. Structure and classification of retroviruses. In The Retroviridae, ed. JA Levy, pp. 19-49. New York: Plenum
30. Coffin JM. 1996. Retroviridae: The Viruses and Their Replication. See Ref. 41a, pp. 1767-848
31. Coffin JM, Hughes SH, Varmus HE, eds. 1997. Retroviruses. New York: Cold Spring Harbor Lab.
32. Cone RD, Mulligan RC. 1984. High-efficiency gene transfer into mammalian cells: Generation of helper-free recombinant retrovirus with broad mammalian host range. Proc. Natl. Acad. Sci. USA 81:6349-53
33. Cullen BR. 1998. HIV-1 auxiliary proteins: Making connections in a dying cell. Cell 93:685-92
34. Daly G, Chernajovsky Y. 2000. Recent developments in retroviral-mediated gene transduction. Mol. Ther. 2:423-34
35. Danos O, Mulligan RC. 1988. Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges. Proc. Natl. Acad. Sci. USA 85:6460-64
36. Dong JY, Fan PD, Frizzell RA. 1996. Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum. Gene Ther. 7:2101-12
37. Duan D, Sharma P, Yang J, Yue Y, Dudus L, et al. 1998. Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue. J. Virol. 72:8568-77
38. Duan D, Yue Y, Yan Z, Engelhardt JF. 2000. A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation. Nat. Med. 6:595-98
39. Dull T, Zufferey R, Kelly M, Mandel RJ, Nguyen M, et al. 1998. A third-generation lentivirus vector with a conditional packaging system. J. Virol. 72:8463-71
40. Emerman M, Malim MH. 1998. HIV-1 regulatory/accessory genes: Keys to unraveling viral and host cell biology. Science 280:1880-84
41. Emerman M, Temin HM. 1984. Genes with promoters in retrovirus vectors can be independently suppressed by an epigenetic mechanism. Cell 39:449-67
42. Engelhardt JF, Ye X, Doranz B, Wilson JM. 1994. Ablation of E2A in recombinant adenoviruses improves transgene persistence and decreases inflammatory response in mouse liver. Proc. Natl. Acad. Sci. USA 91:6196-200
43. Fields BN, Knipe DM, Howley PM, eds. 1996. Fields Virology. Philadelphia: Lippincott-Raven
44. Finer MH, Dull TJ, Qin L, Farson D, Roberts MR. 1994. kat: A high-efficiency retroviral transduction system for primary human T lymphocytes. Blood 83:43-50
45. Follenzi A, Ailles LE, Bakovic S, Geuna M, Naldini L. 2000. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat. Genet. 25:217-22
46. Frenkel N, Singer O, Kwong AD. 1994. Minireview: The herpes simplex virus amplicon - A versatile defective virus vector. Gene Ther 1:S40-46
47. Friedmann T, ed. 1999. The Development of Human Gene Therapy. New York: Cold Spring Harbor Lab.
48. Friedmann T, Roblin R. 1972. Gene therapy for human genetic disease? Science 175:949-55
49. Gallay P, Swingler S, Song J, Bushman F, Trono D. 1995. HIV nuclear import is governed by the phosphotyrosine-mediated binding of matrix to the core domain of integrase. Cell 83:569-76
50. Gao GP, Yang Y, Wilson JM. 1996. Biology of adenovirus vectors with E1 and E4 deletions for liver-directed gene therapy. J. Virol. 70:8934-43
51. Gorman CM, Gies D, McCray G, Huang M. 1989. The human cytomegalovirus major immediate early promoter can be trans-activated by adenovirus early proteins. Virology 171:377-85
52. Gossen M, Bujard H. 1992. Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proc. Natl. Acad. Sci. USA 89:5547-51
53. Gossen M, Freundlieb S, Bender G, Muller G, Hillen W, Bujard H. 1995. Transcriptional activation by tetracyclines in mammalian cells. Science 268:1766-69
54. Graham FL, Smiley J, Russell WC, Nairn R. 1977. Characteristics of a human cell line transformed by DNA from human adenovirus type 5. J. Gen. Virol. 36:59-74
55. Grimm D, Kern A, Rittner K, Kleinschmidt JA. 1998. Novel tools for production and purification of recombinant adeno-associated virus vectors. Hum. Gene Ther. 9:2745-60
56. Grimm D, Kleinschmidt JA. 1999. Progress in adeno-associated virus type 2 vector production: Promises and prospects for clinical use. Hum. Gene Ther. 10:2445-50
57. Gu H, Zou YR, Rajewsky K. 1993. Independent control of immunoglobulin switch recombination at individual switch regions evidenced through Cre-loxP-mediated gene targeting. Cell 73:1155-64
58. Hackett NR, Crystal RG. 2000. Adenovirus vectors for gene therapy. In Gene Therapy, ed. NS Templeton, DD Lasic, pp. 17-39. New York: Marcel Dekker
59. Haj-Ahmad Y, Graham FL. 1986. Development of a helper-independent human adenovirus vector and its use in the transfer of the herpes simplex virus thymidine kinase gene. J. Virol. 57:267-74
60. Hanenberg H, Xiao XL, Dilloo D, Hashino K, Kato I, Williams DA. 1996. Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells. Nat. Med. 2:876-82
61. Hardy S, Kitamura M, Harris-Stansil T, Dai Y, Phipps ML. 1997. Construction of adenovirus vectors through Cre-lox recombination. J. Virol. 71:1842-49
62. Harrison SC, Skehel JJ, Wiley DC. 1996. Virus Structure. See Ref. 41a, pp. 59-99
63. Hearing P, Samulski RJ, Wishart WL, Shenk T. 1987. Identification of a repeated sequence element required for efficient encapsidation of the adenovirus type 5 chromosome. J. Virol. 61:2555-58
64. Heinzinger NK, Bukinsky MI, Haggerty SA, Ragland AM, Kewalramani V, et al. 1994. The Vpr protein of human immunodeficiency virus type 1 influences nuclear localization of viral nucleic acids in nondividing host cells. Proc. Natl. Acad. Sci. USA 91:7311-15
65. Heise C, Sampson-Johannes A, Williams A, McCormick F, Von Hoff DD, Kirn DH. 1997. ONYX-015, an E1B gene-attenuated adenovirus, causes tumor-specific cytolysis and antitumoral efficacy that can be augmented by standard chemotherapeutic agents. Nat. Med. 3:639-45
66. Hermiston T 2000. Gene delivery from replication-selective viruses: Arming guided missiles in the war against cancer. J. Clin. Invest. 105:1169-72
67. Hirsch MS, Curran J. 1996. Human Immunodeficiency Viruses. See Ref. 41a, pp. 1953-75
68. Hitt MM, Parks RJ, Graham FL. 1999. Structure and Genetic Organization of Adenovirus Vectors. See Ref. 44a, pp. 61-86
69. Hofmann A, Nolan GP, Blau HM. 1996. Rapid retroviral delivery of tetracycline-inducible genes in a single autoregulatory cassette. Proc. Natl. Acad. Sci. USA 93:5185-90
70. Horwitz MS. 1996. Adenoviruses. See Ref. 41a, pp. 2149-71
71. Huang HV. 1996. Sindbis virus vectors for expression in animal cells. Curr. Opin. Biotechnol. 7:531-35
72. Inoue N, Russell DW. 1998. Packaging cells based on inducible gene amplification for the production of adeno-associated virus vectors. J. Virol. 72:7024-31
73. Joag SV, Stephens EB, Narayan O. 1996. Lentiviruses. See Ref. 41a, pp. 1977-96
74. Jolly DJ. 1999. Emerging Viral Vectors. See Ref. 44a, pp. 209-40
75. Kafri T, Blomer U, Peterson DA, Gage FH, Verma IM. 1997. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat. Genet. 17:314-17
76. Kafri T, Morgan D, Krahl T, Sarvetnick N, Sherman L, Verma I. 1998. Cellular immune response to adenoviral vector infected cells does not require de novo viral gene expression: Implications for gene therapy. Proc. Natl. Acad. Sci. USA 95:11377-82
77. Kafri T, van Praag H, Ouyang L, Gage FH, Verma IM. 1999. A packaging cell line for lentivirus vectors. J. Virol. 73:576-84
78. Kafri T, von Praag H, Gage FH, Verma IM. 2000. Lentiviral vectors - Regulated gene expression. Mol. Ther. 1:516-21
79. Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, et al. 2000. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat. Genet. 24:257-61
80. Ketner G, Spencer F, Tugendreich S, Connelly C, Hieter P. 1994. Efficient manipulation of the human adenovirus genome as an infectious yeast artificial chromosome clone. Proc. Natl. Acad. Sci. USA 91:6186-90
81. Khuri FR, Nemunaitis J, Ganly I, Arseneau J, Tannock IF, et al. 2000. A controlled trial of intratumoral ONYX-015, a selectively-replicating adenovirus, in combination with cisplatin and 5-fluorouracil in patients with recurrent head and neck cancer. Nat. Med. 6:879-85
82. Kiem HP, Andrews RG, Morris J, Peterson L, Heyward S, et al. 1998. Improved gene transfer into baboon marrow repopulating cells using recombinant human fibronectin fragment CH-296 in combination with interleukin-6, stem cell factor, FLT-3 ligand, and megakaryocyte growth and development factor. Blood 92:1878-86
83. Klages N, Zufferey R, Trono D. 2000. A stable system for the high-titer production of multiply attenuated lentiviral vectors. Mol. Ther. 2:170-76
84. Klessig DF, Brough DE, Cleghon V. 1984. Introduction, stable integration, and controlled expression of a chimeric adenovirus gene whose product is toxic to the recipient human cell. Mol. Cell Biol. 4:1354-62
85. Kochanek S. 1999. High-capacity adenoviral vectors for gene transfer and somatic gene therapy. Hum. Gene Ther. 10:2451-59
86. Kochanek S, Clemens PR, Mitani K, Chen HH, Chan S, Caskey CT. 1996. A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase. Proc. Natl. Acad. Sci. USA 93:5731-36
87. Krisky DM, Marconi PC, Oligino TJ, Rouse RJ, Fink DJ, et al. 1998. Development of herpes simplex virus replication-defective multigene vectors for combination gene therapy applications. Gene Ther. 5:1517-30
88. Krougliak V, Graham FL. 1995. Development of cell lines capable of complementing E1, E4, and protein IX defective adenovirus type 5 mutants. Hum. Gene Ther. 6:1575-86
89. Leboulch P, Huang GM, Humphries RK, Oh YH, Eaves CJ, et al. 1994. Mutagenesis of retroviral vectors transducing human beta-globin gene and beta-globin locus control region derivatives results in stable transmission of an active transcriptional structure. EMBO J. 13:3065-76
90. Lee HC, Kim SJ, Kim KS, Shin HC, Yoon JW. 2000. Remission in models of type 1 diabetes by gene therapy using a single-chain insulin analogue. Nature 408:483-88
91. Li J, Samulski RJ, Xiao X. 1997. Role for highly regulated rep gene expression in adeno-associated virus vector production. J. Virol. 71:5236-43
92. Lieber A, He CY, Kirillova I, Kay MA. 1996. Recombinant adenoviruses with large deletions generated by Cre-mediated excision exhibit different biological properties compared with first-generation vectors in vitro and in vivo. J. Virol. 70:8944-60
93. Lindemann D, Patriquin E, Feng S, Mulligan RC. 1997. Versatile retrovirus vector systems for regulated gene expression in vitro and in vivo. Mol. Med. 3:466-76
94. Loeb JE, Cordier WS, Harris ME, Weitzman MD, Hope TJ. 1999. Enhanced expression of transgenes from adeno-associated virus vectors with the woodchuck hepatitis virus posttranscriptional regulatory element: Implications for gene therapy. Hum. Gene Ther. 10:2295-305
95. Mangeot PE, Negre D, Dubois B, Winter AJ, Leissner P, et al. 2000. Development of minimal lentivirus vectors derived from simian immunodeficiency virus (SIVmac251) and their use for gene transfer into human dendritic cells. J. Virol. 74:8307-15
96. Mann R, Mulligan RC, Baltimore D. 1983. Construction of a retrovirus packaging mutant and its use to produce helper-free defective retrovirus. Cell 33:153-59
97. Markowitz D, Goff S, Bank A. 1988. A safe packaging line for gene transfer: Separating viral genes on two different plasmids. J. Virol. 62:1120-24
98. Mastromarino P, Conti C, Goldoni P, Hauttecoeur B, Orsi N. 1987. Characterization of membrane components of the erythrocyte involved in vesicular stomatitis virus attachment and fusion at acidic pH. J. Gen. Virol. 68:2359-69
99. May C, Rivella S, Callegari J, Heller G, Gaensler KM, et al. 2000. Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin. Nature 406:82-86
100. McCown TJ, Xiao X, Li J, Breese GR, Samulski RJ. 1996. Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector. Brain Res. 713:99-107
101. McGrory WJ, Bautista DS, Graham FL. 1988. A simple technique for the rescue of early region I mutations into infectious human adenovirus type 5. Virology 163:614-17
102. McLaughlin SK, Collis P, Hermonat PL, Muzyczka N. 1988. Adeno-associated virus general transduction vectors: Analysis of proviral structures. J. Virol. 62:1963-73
103. Miller AD. 1997. Development and Application of Retroviral Vectors. See Ref. 30a, pp. 437-73
104. Miller AD, Buttimore C. 1986. Redesign of retrovirus packaging cell lines to avoid recombination leading to helper virus production. Mol. Cell Biol. 6:2895-902
105. Miller AD, Law MF, Verma IM. 1985. Generation of helper-free amphotropic retroviruses that transduce a dominant-acting, methotrexate-resistant dihydrofolate reductase gene. Mol. Cell Biol. 5:431-37
106. Miller DG, Adam MA, Miller AD. 1990. Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol. Cell Biol. 10:4239-42. Erratum. 1992. Mol. Cell Biol. 12(1):433
107. Mineta T, Rabkin SD, Yazaki T, Hunter WD, Martuza RL. 1995. Attenuated multimutated herpes simplex virus 1 for the treatment of malignant gliomas. Nat. Med. 1:938-43
108. Miyoshi H, Blomer U, Takahashi M, Gage FH, Verma IM. 1998. Development of a self-inactivating lentivirus vector. J. Virol. 72:8150-57
109. + cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science 283:682-86
110. Miyoshi H, Takahashi M, Gage FH, Verma IM. 1997. Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector. Proc. Natl. Acad. Sci. USA 94:10319-23
111. Monahan PE, Samulski RJ. 2000. AAV vectors: Is clinical success on the horizon? Gene Ther. 7:24-30
112. Moritz T, Dutt P, Xiao X, Carstanjen D, Vik T, et al. 1996. Fibronectin improves transduction of reconstituting hematopoietic stem cells by retroviral vectors: Evidence of direct viral binding to chymotryptic carboxy-terminal fragments. Blood 88:855-62
113. Muzyczka N. 1992. Use of adeno-associated virus as a general transduction vector for mammalian cells. Curr. Top. Microbiol. Immunol. 158:97-129
114. Nakai H, Iwaki Y, Kay MA, Couto LB. 1999. Isolation of recombinant adeno-associated virus vector - Cellular DNA junctions from mouse liver. J. Virol. 73:5438-47
115. Nakai H, Storm TA, Kay MA. 2000. Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors. Nat. Biotechnol. 18:527-32
116. Naldini L, Blomer U, Gage FH, Trono D, Verma IM. 1996. Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc. Natl. Acad. Sci. USA 93:11382-88
117. Naidini L, Blomer U, Gallay P, Ory D, Mulligan R, et al. 1996. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272:263-67
118. Naldini L, Verma IM. 1999. Lentiviral Vectors. See Ref. 44a, pp. 47-60
119. Naviaux RK, Costanzi E, Haas M, Verma IM. 1996. The pCL vector system: Rapid production of helper-free, hightiter, recombinant retroviruses. J. Virol. 70:5701-5
120. No D, Yao TP, Evans RM. 1996. Ecdysone-inducible gene expression in mammalian cells and transgenic mice. Proc. Natl. Acad. Sci. USA 93:3346-51
121. Nolta JA, Smogorzewska EM, Kohn DB. 1995. Analysis of optimal conditions for retroviral-mediated transduction of primitive human hematopoietic cells. Blood 86:101-10
122. Olsen JC. 1998. Gene transfer vectors derived from equine infectious anemia virus. Gene Ther. 5:1481-87
123. Ory DS, Neugeboren BA, Mulligan RC. 1996. A stable human-derived packaging cell line for production of high titer retrovirus/vesicular stomatitis virus G pseudotypes. Proc. Natl. Acad. Sci. USA 93:11400-6
124. Parks RJ, Chen L, Anton M, Sankar U, Rudnicki MA, Graham FL. 1996. A helper-dependent adenovirus vector system: Removal of helper virus by Cre-mediated excision of the viral packaging signal. Proc. Natl. Acad. Sci. USA 93:13565-70
125. Parks RJ, Graham FL. 1997. A helper-dependent system for adenovirus vector production helps define a lower limit for efficient DNA packaging. J. Virol. 71:3293-98
126. Pfeifer A, Kessler T, Yang M, Baranov E, Kootstra NA, et al. 2001. Transduction of liver cells by lentiviral vectors: Analysis in living animals by fluorescence imaging. Mol. Ther. 3:319-22
127. Pfeifer A, Verma IM. 2001. Virus vectors and their applications. In Fields Virology, ed. PM Howley, DM Knipe, D Griffin, RA Lamb, A Martin, et al. Philadelphia: Lippincott-Raven. In press
128. Poeschla E, Corbeau P, Wong-Staal F. 1996. Development of HIV vectors for anti-HIV gene therapy. Proc. Natl. Acad. Sci. USA 93:11395-99
129. Poeschla E, Gilbert J, Li X, Huang S, Ho A, Wong-Staal F. 1998. Identification of a human immunodeficiency virus type 2 (HIV-2) encapsidation determinant and transduction of nondividing human cells by HIV-2-based lentivirus vectors. J. Virol. 72:6527-36
130. Poeschla EM, Wong-Staal F, Looney DJ. 1998. Efficient transduction of nondividing human cells by feline immunodeficiency virus lentiviral vectors. Nat. Med. 4:354-57
131. Pollock R, Issner R, Zoller K, Natesan S, Rivera VM, Clackson T. 2000. Delivery of a stringent dimerizer-regulated gene expression system in a single retroviral vector. Proc. Natl. Acad. Sci. USA 97:13221-26
132. Pollock R, Rivera VM. 1999. Regulation of gene expression with synthetic dimerizers. Methods Enzymol. 306:263-81
133. Poznansky M, Lever A, Bergeron L, Haseltine W, Sodroski J. 1991. Gene transfer into human lymphocytes by a defective human immunodeficiency virus type 1 vector. J. Virol. 65:532-36
134. Qing K, Mah C, Hansen J, Zhou S, Dwarki V, Srivastava A. 1999. Human fibroblast growth factor receptor 1 is a coreceptor for infection by adeno-associated virus 2. Nat. Med. 5:71-77
135. Rabson AB, Wills JW. 1997. Synthesis and Processing of Viral RNA. See Ref. 30a, pp. 205-61
136. Ramirez-Solis R, Liu P, Bradley A. 1995. Chromosome engineering in mice. Nature 378:720-24
137. Reiser J, Harmison G, Kluepfel-Stahl S, Brady RO, Karlsson S, Schubert M. 1996. Transduction of nondividing cells using pseudotyped defective high-titer HIV type 1 particles. Proc. Natl. Acad. Sci. USA 93:15266-71
138. Rivera VM, Clackson T, Natesan S, Pollock R, Amara JF, et al. 1996. A humanized system for pharmacologic control of gene expression. Nat. Med. 2:1028-32
139. Roizman B, Sears AE. 1996. Adenoviruses. See Ref. 41a, pp. 2231-95
140. Saeki Y, Ichikawa T, Saeki A, Chiocca EA, Tobler K, et al. 1998. Herpes simplex virus type 1 DNA amplified as bacterial artificial chromosome in Escherichia coli: Rescue of replication-competent virus progeny and packaging of amplicon vectors. Hum. Gene Ther. 9:2787-94
141. Samaniego LA, Neiderhiser L, DeLuca NA. 1998. Persistence and expression of the herpes simplex virus genome in the absence of immediate-early proteins. J. Virol. 72:3307-20
142. Samulski RJ, Chang LS, Shenk T. 1987. A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication. J. Virol. 61:3096-101
143. Samulski RJ, Chang LS, Shenk T. 1989. Helper-free stocks of recombinant adeno-associated viruses: Normal integration does not require viral gene expression. J. Virol. 63:3822-28
144. Samulski RJ, Sally M, Muzyczka N. 1999. Adeno-Associated Viral Vectors. See Ref. 44a, pp. 131-72
145. Sarkis C, Serguera C, Petres S, Buchet D, Ridet JL, et al. 2000. Efficient transduction of neural cells in vitro and in vivo by a baculovirus-derived vector. Proc. Natl. Acad. Sci. USA 97:14638-43
146. Shenk T. 1996. Adenoviruses: The Viruses and Their Replication. See Ref. 41a, pp. 2111-48
147. 4+ cells by a recombinant human immunodeficiency virus retroviral vector. J. Clin. Invest. 88:1043-47
148. Shockett P, Difilippantonio M, Hellman N, Schatz DG. 1995. A modified tetracycline-regulated system provides autoregulatory, inducible gene expression in cultured cells and transgenic mice. Proc. Natl. Acad. Sci. USA 92:6522-26
149. Snyder RO, Miao CH, Patijn GA, Spratt SK, Danos O, et al. 1997. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat. Genet. 16:270-76
150. Somia N, Verma IM. 2001. Gene therapy: Trials and tribulations. Nat. Rev. Genet. 1:91-99
151. Spector DH, Wade E, Wright DA, Koval V, Clark C, et al. 1990. Human immunodeficiency virus pseudotypes with expanded cellular and species tropism. J. Virol. 64:2298-308
152. Srivastava A. 1994. Parvovirus-based vectors for human gene therapy. Blood Cells 20:531-36
153. Strauss EG, Strauss JH, Levine AJ. 1996. Virus Evolution. See Ref. 41a, pp. 153-71
154. Suhr ST, Gil EB, Senut MC, Gage FH. 1998. High level transactivation by a modified Bombyx ecdysone receptor in mammalian cells without exogenous retinoid X receptor. Proc. Natl. Acad. Sci. USA 95:7999-8004
155. Summerford C, Bartlett JS, Samulski RJ. 1999. αV β5 integrin: A co-receptor for adeno-associated virus type 2 infection. Nat. Med. 5:78-82
156. Summerford C, Samulski RJ. 1998. Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J. Virol. 72:1438-45
157. Sun L, Li J, Xiao X. 2000. Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization. Nat. Med. 6:599-602
158. Takahashi M, Miyoshi H, Verma IM, Gage FH. 1999. Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer. J. Virol. 73:7812-16
159. Telesnitsky A, Goff SP. 1997. Reverse Transcriptase and the Generation of Retroviral DNA. See Ref. 30a, pp. 121-60
160. Tisdale JF, Hanazono Y, Sellers SE, Agricola BA, Metzger ME, et al. 1998. Ex vivo expansion of genetically marked rhesus peripheral blood progenitor cells results in diminished long-term repopulating ability. Blood 92:1131-41
161. Trono D. 2000. Lentiviral vectors: Turning a deadly foe into a therapeutic agent. Gene Ther. 7:20-23
162. US Dept. Health. 1999. Minutes of Symp. Meet., Recombinant DNA Advisory Comm., Dec. 8-10. Washington, DC: Natl. Inst. Health. http://www4.od.nih.gov/oba/1299rac.pdf
163. Verma IM, Somia N. 1997. Gene therapy - Promises, problems and prospects. Nature 389:239-42
164. Vogt PK. 1997. Historical Introduction to the General Properties of Retroviruses. See Ref. 30a, pp. 1-25
165. Wang Q, Finer MH. 1996. Second-generation adenovirus vectors. Nat. Med. 2:714-16
166. Wang Q, Greenburg G, Bunch D, Farson D, Finer MH. 1997. Persistent transgene expression in mouse liver following in vivo gene transfer with a deltaE1/deltaE4 adenovirus vector. Gene Ther. 4:393-400
167. Wang Q, Jia XC, Finer MH. 1995. A packaging cell line for propagation of recombinant adenovirus vectors containing two lethal gene-region deletions. Gene Ther. 2:775-83
168. Wang Y, O'Malley BW Jr, Tsai SY, O'Malley BW. 1994. A regulatory system for use in gene transfer. Proc. Natl. Acad. Sci. USA 91:8180-84
169. Weinberg JB, Matthews TJ, Cullen BR, Malim MH. 1991. Productive human immunodeficiency virus type 1 (HIV-1) infection of nonproliferating human monocytes. J. Exp. Med. 174:1477-82
170. Weitzman MD, Kyostio SR, Kotin RM, Owens RA. 1994. Adeno-associated virus (AAV) Rep proteins mediate complex formation between AAV DNA and its integration site in human DNA. Proc. Natl. Acad. Sci. USA 91:5808-12
171. Wickham TJ, Mathias P, Cheresh DA, Nemerow GR. 1993. Integrins αV β3 and αV β5 promote adenovirus internalization but not virus attachment. Cell 73:309-19
172. Wivel NA, Gao G-P, Wilson JM. 1999. Adenovirus Vectors. See Ref. 44a, pp. 87-111
173. Wu P, Phillips MI, Bui J, Terwilliger EF. 1998. Adeno-associated virus vector-mediated transgene integration into neurons and other nondividing cell targets. J. Virol. 72:5919-26
174. Xiao X, Li J, Samulski RJ. 1996. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J. Virol. 70:8098-108
175. Xiao X, Li J, Samulski RJ. 1998. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J. Virol. 72:2224-32
176. Xu K, Ma H, McCown TJ, Verma IM, Kafri T. 2001. Generation of a stable cell line producing high-titer self-inactivating lentiviral vectors. Mol. Ther. 3:97-104
177. Yan Z, Zhang Y, Duan D, Engelhardt JF. 2000. From the cover: Transsplicing vectors expand the utility of adeno-associated virus for gene therapy. Proc. Natl. Acad. Sci. USA 97:6716-21
178. Yang CC, Xiao X, Zhu X, Ansardi DC, Epstein ND, et al. 1997. Cellular recombination pathways and viral terminal repeat hairpin structures are sufficient for adeno-associated virus integration in vivo and in vitro. J. Virol. 71:9231-47
179. Yee J-K. 1999. Retroviral Vectors. See Ref. 44a, pp. 21-46
180. Yee JK, Moores JC, Jolly DJ, Wolff JA, Respess JG, Friedmann T. 1987. Gene expression from transcriptionally disabled retroviral vectors. Proc. Natl. Acad. Sci. USA 84:5197-201
181. Yeh P, Dedieu JF, Orsini C, Vigne E, Denefle P, Perricaudet M. 1996. Efficient dual transcomplementation of adenovirus E1 and E4 regions from a 293-derived cell line expressing a minimal E4 functional unit. J Virol 70:559-65
182. Yew PR, Berk AJ. 1992. Inhibition of p53 transactivation required for transformation by adenovirus early 1B protein. Nature 357:82-85
183. Young NS. 1996. Parvoviruses. See Ref. 41a, pp. 2199-220
184. Yu H, Rabson AB, Kaul M, Ron Y, Dougherty JP. 1996. Inducible human immunodeficiency virus type 1 packaging cell lines. J. Virol. 70:4530-37
185. Yu SF, von Ruden T, Kantoff PW, Garber C, Seiberg M, et al. 1986. Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells. Proc. Natl. Acad. Sci. USA 83:3194-98
186. Zennou V, Petit C, Guetard D, Nerhbass U, Montagnier L, Charneau P. 2000. HIV-1 genome nuclear import is mediated by a central DNA flap. Cell 101:173-85
187. Zolotukhin S, Byrne BJ, Mason E, Zolotukhin I, Potter M, et al. 1999. Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther. 6:973-85
188. Zufferey R, Donello JE, Trono D, Hope TJ. 1999. Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J. Virol. 73:2886-92
189. Zufferey R, Dull T, Mandel RJ, Bukovsky A, Quiroz D, et al. 1998. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J. Virol. 72:9873-80
190. Zufferey, R, Nagy D, Mandel RJ, Naldini L, Trono D. 1997. Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nat. Biotechnol. 15:871-75