A concise peer into the background, initial thoughts and practices of human gene therapy

BioEssays

Volumn 27, Issue 5, 2005, Pages 506-517

  Gonçalves, Manuel A F V ^a  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; PARVOVIRUS VECTOR; RECOMBINANT DNA; RETROVIRUS VECTOR;

ALLELE; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENE INSERTION; GENE ISOLATION; GENE TECHNOLOGY; GENE THERAPY; GENE TRANSFER; GENETIC DISORDER; HUMAN; HUMAN GENETICS; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; RECOMBINANT GENE; REVIEW; VIRAL GENE DELIVERY SYSTEM; ANIMAL; GENE VECTOR; GENETICS; RETROVIRUS;

ADENO-ASSOCIATED VIRUS; PRIMATES;

ANIMALS; DNA, RECOMBINANT; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; RETROVIRIDAE;

EID: 18744391982     PISSN: 02659247     EISSN: None     Source Type: Journal    
DOI: 10.1002/bies.20218     Document Type: Review

References (100)

1. Friedmann T. 1996. Human gene therapy-an immature genie, but certainly out of the bottle. Nat Med 2:144-147.
2. Williams ML, Koch WJ. 2004. Viral-based myocardial gene therapy approaches to alter cardiac function. Annu Rev Physiol 66:49-75.
3. Gottesman MM. 2003. Cancer gene therapy: an awkward adolescence. Cancer Gene Ther 10:501-508.
4. Fanning G, Amado R, Symonds G. 2003. Gene therapy for HIV/AIDS: the potential for a new therapeutic regimen. J Gene Med 5:645-653.
5. Welch PJ, Barber JR, Wong-Staal F. 1998. Expression of ribozymes in gene transfer systems to modulate target RNA levels. Curr Opin Biotechnol 9:486-496.
6. Xia H, Mao Q, Eliason SL, Harper SQ, Martins IH, et al. 2004. RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. Nat Med 10:816-820.
7. Chamberlain JR, Schwarze U, Wang P-R, Hirata RK, Hankenson KD, et al. 2004. Gene targeting in stem cells from individuals with osteogenesis imperfecta. Science 303:1198-1201.
8. Stevenson M. 2004. Therapeutic potential of RNA interference. N Engl J Med 351:1772-1777.
9. Kay LE. 1993. The Molecular Vision of Life: Caltech, The Rockefeller Foundation, and the Rise of the New Biology. In: Burian R, Burkhardt Jr R, Lewontin R, Smith JM, editors. Monographs on the History and Philosophy of Biology. New York, NY: Oxford University Press.
10. Zinder ND, Lederberg J. 1952. Genetic exchange in Salmonella. J Bacteriol 64:679-699.
11. Lwoff A. 1953. Lysogeny. Bacteriol Rev 17:269-337.
12. Campbell AM. 1993. Thirty years ago in genetics: prophage insertion into bacterial chromosomes. Genetics 133:433-438.
13. Temin HM. 1961. Mixed infection with two types of Rous sarcoma virus. Virology 13:158-163.
14. Crawford LV, Crawford EM. 1961. The properties of Rous sarcoma virus purified by density gradient centrifugation. Virology 23:227-232.
15. Baltimore D. 1970. RNA-dependent DNA polymerase in virions of RNA tumor viruses. Nature 226:1209-1211.
16. Temin HM, Mizutani S. 1970. RNA-dependent DNA polymerase in virions of Rous sarcoma virus. Nature 226:1211-1213.
17. Neiman PE. 1972. Rous sarcoma virus nucleotide sequences in cellular DNA: measurement of RNA-DNA hybridisation. Science 178:750-753.
18. Sambrook J, Westphal H, Srinivasan PR, Dulbecco R. 1968. The integrated state of viral DNA in SV40-transformed cells. Proc Natl Acad Sci USA 60:1288-1295.
19. Tatum EL 1966. Molecular biology, nucleic acids and the future of medicine. Perspect Biol Med 10:19-32.
20. Rogers S. 1966. Shope papilloma virus: a passenger in man and its significance to the potential control of the host genome. Nature 212:1220-1222.
21. Medawar PB, Medawar JS. 1983. Viruses. In: Medawar PB, Medawar JS, editors. Aristotle to zoos: a philosophical dictionary of biology. Cambridge, MA: Harvard University Press, p 275.
22. Rogers S, Pfuderer P. 1968. Use of viruses as carriers of added genetic information. Nature 219:749-751.
23. Rogers S. 1959. Induction of arginase in rabbit epithelium by the Shope papilloma virus. Nature 183:1815-1817.
24. Orth G, Vielle F, Changeux JP. 1967. On the arginase of the Shope pappolas. Virology 31:729-732.
25. Satoh PS, Yoshida TO, Ito Y. 1967. Studies on the arginase activity of Shope papilloma: possible presence of isozymes. Virology 33:354-356.
26. Girl I, Danos O, Yaniv M. 1985. Genomic structure of the cottontail rabbit (Shope) papillomavirus. Proc Natl Acad Sci USA 82:1580-1584.
27. Munyon W, Kraiselburd E, Davis D, Mann J. 1971. Transfer of thymidine kinase to thymidine kinaseless L cells by infection with ultraviolet-irradiated herpes simplex virus. J Virol 7:813-820.
28. Hershey AD, Burgi E. 1965. Complementary structure of interacting sites at the ends of lambda DNA molecules. Proc Natl Acad Sci USA 53:325-328.
29. Watson JD, Gilman G, Witkowski J, Zoller M. 1992. Methods of creating recombinant DNA molecules. In Recombinant DNA, 2nd Edition. New York: Scientific American Books, p 63-77.
30. Berg P, Baltimore D, Boyer HW, Cohen SN, Davis RW, et al. 1974. Potential biohazards of recombinant DNA molecules. Science 185:303.
31. Smith HO, Wilcox KW. 1970. A restriction enzyme from Hemophilus influenzae. I. Purification and general properties. J Mol Biol 51:379-391.
32. Danna K, Nathans D. 1971. Specific cleavage of simian virus 40 DNA by restriction endonuclease of Hemophilus influenzae. Proc Natl Acad Sci USA 68:2913-2917.
33. Luria SE, Human ML. 1952. A nonhereditary, host-induced variation of bacterial viruses. J Bacteriol 64:557-569.
34. Arber W. 1965. Host-controlled modification of bacteriophage. Annu Rev Microbiol 19:365-378.
35. Mertz JE, Berg P. 1974. Defective simian virus 40 genomes: isolation and growth of individual clones. Virology 62:112-124.
36. Goff SP, Berg P. 1976. Construction of hybrid viruses containing SV40 and lambda phage DNA segments and their propagation in cultured monkey cells. Cell 9:695-705.
37. Goff SP, Berg P. 1979. Construction, propagation and expression of simian virus 40 recombinant genomes containing the Escherichia coli gene for thymidine kinase and a Saccharomyces cerevisae gene for tyrosine transfer RNA. J Mol Biol 133:359-383.
38. Ganem D, Nussbaum AL, Davoli D, Fareed GC. 1976. Propagation of a segment of bacteriophage λ-DNA in monkey cells after covalent linkage to a defective simian virus 40 genome. Cell 7:349-359.
39. Hamer DH, Davoli D, Thomas CA, Fareed GC. 1977. Simian virus 40 carrying an Escherichia coli suppressor gene. J Mol Biol 112:155-182.
40. Mulligan RC, Howard BH, Berg P. 1979. Synthesis of rabbit β-globin in cultured monkey kidney cells following infection with a SV40 β-globin recombinant genome. Nature 277:108-114.
41. Hamer DH, Smith KD, Boyer SH, Leder P. 1979. SV40 recombinants carrying rabbit β-globin gene coding sequences. Cell 17:725-736.
42. Maniatis T, Kee SG, Efstratiadis A, Kafatos FC. 1976. Amplification and characterization of a β-globin gene synthesized in vitro. Cell 8:163-182.
43. maj-globin gene cloned in SV40. Nature 281:35-40.
44. Hamer DH, Kaehler M, Leder P. 1980. A mouse globin gene promoter is functional in SV40. Cell 21:697-708.
45. Mulligan RC, Berg P. 1980. Expression of a bacterial gene in mammalian cells. Science 209:1422-1427.
46. Szybalski EH, Szybalski W. 1962. Genetics of human cell lines, IV. DNA-mediated heritable transformation of a biochemical trait. Proc Natl Acad Sci USA 48:2026-2034.
47. Szybalski W. 1992. Use of the HPRT gene and the HAT selection technique in DNA-mediated transformation of mammalian cells: first steps toward developing hybridoma techniques and gene therapy. Bioessays 14:495-500.
48. Graham FL, van der Eb A. 1973. A new technique for the assay of infectivity of human adenovirus 5 DNA. Virology 52:456-467.
49. Vaheri A, Pagano JS. 1965. Infectious poliovirus RNA: a sensitive method of assay. Virology 27:434-436.
50. Cline MJ, Stang H, Mercola K, Morse L, Ruprecht R, et al. 1980. Gene transfer in intact animals. Nature 284:422-425.
51. Mercola KE, Stang HD, Browne J, Salser W, Cline MJ. 1980. Insertion of a new gene of viral origin into BM cells of mice. Science 208:1033-1035.
52. Shimotohno K, Temin HM. 1981. Formation of infectious progeny virus after insertion of herpes simplex thymidine kinase gene into DNA of an avian retrovirus. Cell 26:67-77.
53. Wei C-M, Gibson M, Spear PG, Scolnick EM. 1981. Construction and isolation of a transmissible retrovirus containing the src gene of Harvey murine sarcoma virus and the thymidine kinase gene of herpes simplex virus type 1. J Virol 39:935-944.
54. Tabin CJ, Hoffmann JW, Goff SP, Weinberg RA. 1982. Adaptation of a retrovirus as a eucaryotic vector transmitting the herpes simplex virus thymidine kinase gene. J Mol Biol 2:426-436.
55. Miller AD. 1990. Retrovirus packaging cells. Hum Gene Ther 1:5-14.
56. Miller AD, Jolly DJ, Friedmann T, Verma IM. 1983. A transmissible retrovirus expressing human hypoxanthine phosphoribosyltransferase (HPRT): gene transfer into cells obtained from humans deficient in HPRT. Proc Natl Acad Sci USA 80:4709-4713.
57. Joyner A, Keller G, Phillips RA, Bernstein A. 1983. Retrovirus transfer of a bacterial gene into mouse haematopoietic progenitor cells. Nature 305:556-558.
58. Williams DA, Lemischka IR, Nathan DG, Mulligan RC. 1984. Introduction of new genetic material into pluripotent haematopoietic stem cells of the mouse. Nature 310:476-480.
59. v mice. Cell 42:71-79.
60. Eglitis MA, Kantoff P, Gilboa E, Anderson WF. 1985. Gene expression in mice after high efficiency retroviral-mediated gene transfer. Science 230:1395-1398.
61. Keller G, Paige C, Gilboa E, Wagner EF. 1985. Expression of a foreign gene in myeloid and lymphoid cells derived from multipotent haematopoietic precursors. Nature 318:149-154.
62. Lemischka IR, Raulet DH, Mulligan RC. 1986. Developmental potential and dynamic behavior of haematopoietic stem cells. Cell 45:917-927.
63. Wu AM, Till JE, Siminovitch L, McCulloch EA. 1968. Cytological evidence for a relationship between normal hematopoietic colony-forming cells and cells of the immune system. J Exp Med 127:455-463.
64. Hodgson GS, Bradley TR. 1979. Properties of haematopoietic stem cells surviving 5-fluorouracil treatment: evidence for a pre-CFU-S cell? Nature 281:381-382.
65. Metcalf D. 1989. The molecular control of cell division, differentiation commitment and maturation in haemopoietic cells. Nature 339:27-30.
66. Dzierzak EA, Papayannopoulou T, Mulligan RC. 1988. Lineage specific expression of a human β-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells. Nature 331:35-41.
67. Lim B, Apperly JF, Orkin SH, Williams DA, 1989. Long-term expression of human adenosine deaminase in mice transplanted with retrovirus infected hematopoietic stem cells. Proc Natl Acad Sci USA 86:8892-8896.
68. Correll PH, Kew Y, Perry LK, Brady RO, Fink JK, et al. 1990. Expression of human glucocerebrosidase in long-term reconstituted mice following retroviral-mediated gene transfer into hematopoietic stem cells. Hum Gene Ther 1:277-283.
69. Wolfe JH, Sands MS, Barker JE, Gwynn B, Rowe LB, et al. 1992. Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer. Nature 360:749-753.
70. Bodine DM, McDonagh KT, Brandt SI, Ney PA, Agricola B. 1990. Development of a high-liter retrovirus producer cell line capable of gene transfer into rhesus monkey hematopoietic stem cells. Proc Natl Acad Sci USA 87:3738-3742.
71. van Beusechem VW, Kukler A, Heidt PJ, Valerio D. 1992. Long-term expression of human adenosine deaminase in rhesus monkeys transplanted with retrovirus-infected bone marrow cells. Proc Natl Acad Sci USA 89:7640-7646.
72. + bone marrow cells. Blood 82:1975-1980.
73. Bienzle D, Abrams-Ogg AC, Kruth SA, Ackland-Snow J, Carter RF, et al. 1994. Gene transfer into hematopoietic stem cells: long-term maintenance of in vitro activated progenitors without marrow ablation. Proc Natl Acad Sci USA 91:350-354.
74. Rosenberg SA, Aebersold P, Cornelia K, Kasid A, Morgan RA, et al. 1990. Gene transfer into humans-immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med 323:570-578.
75. Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T. 1995. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science 270:475-480.
76. Brenner MK, Rill DR, Moen RC, Krance RA, Mirro Jr J, et al. 1993. Gene-marking to trace origin of relapse after autologous bone-marrow transplantation. Lancet 341:85-86.
77. Miller DG, Adam MA, Miller AD. 1990. Gene transfer by retrovirus H vectors occurs only in cells that are actively replicating at the time of infection. Mol Cell Biol 10:4239-4242.
78. Kiem HP, Heyward S, Winkler A, Potter J, Allen JM. 1997. Gene transfer into marrow repopulating cells: comparison between amphotropic and gibbon ape leukemia virus pseudotyped retroviral vectors in a competitive repopulation assay in baboons. Blood 90:4638-4645.
79. Chien ML, Foster JL, Douglas JL, Garcia JV. 1997. The amphotropic murine leukemia receptor gene encodes a 71-kilodalton protein that is induced by phosphate depletion. J Virol 71:4564-4570.
80. + cells. Hum Gene Ther 9:2727-2734.
81. + cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science 283:682-686.
82. Cockrell AS, Kafri T. 2003. HIV-1 vectors: fulfillment of expectations, further advancements, and still a way to go. Curr HIV Res 1:419-439.
83. van Doren K, Hanahan D, Gluzman Y. 1984. Infection of eukaryotic cells by helper-independent recombinant adenoviruses: early region 1 is not obligatory for integration of viral DNA. J Virol 50:606-614.
84. Hermonat PL, Muzyczka N. 1984. Use of adeno-associated virus as a mammalian DNA cloning vector: tranduction of neomycin resistance into mammalian tissue cultured cells. Proc Natl Acad Sci USA 81:6466-6470.
85. Tratschin JD, West MH, Sandbank T, Carter BJ. 1984. A human parvovirus, adeno-associated virus, as a eukaryotic vector: transient expression and encapsidation of the prokaryotic gene for chloramphenicol acetyltransferase. Mol Cell Biol 4:2072-2081.
86. Crystal RG, Jaffe A, Brody S, Mastrangeli A, McElvaney NG, et al. 1995. A phase I study, in cystic fibrosis patients, of the safety, toxicity, and biological efficacy of a single administration of a replication deficient, recombinant adenovirus carrying the cDNA of the normal cystic fibrosis transmembrane conductance regulator gene in the lung. Hum Gene Ther 6:643-666.
87. Flotte T, Carter B, Conrad C, Guggino W, Reynolds T, et al. 1996. A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum Gene Ther 7:1145-1159.
88. Lai CM, Lai YK, Rakoczy PE. 2002. Adenovirus and adeno-associated virus vectors. DNA Cell Biol 21:895-913.
89. Thomas CE, Ehrhardt A, Kay MA. 2003. Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet 4:346-358.
90. Schmidt-Wolf GD, Schmid-Wolf IG. 2003. Non-viral and hybrid vectors in human gene therapy: an update. Trends Mol Med 9:67-72.
91. Raper SE, Chirmule N, Lee FS, Wivel NA, Bagg A, et al. 2003. Fatal systemic inflammatory response in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab 80:148-158.
92. 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 288:669-672.
93. Aiuti A, Slavin S, Aker M, Ficara F, Deola S, et al. 2002. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 296:2410-2413.
94. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, et al. 2003. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302:415-419.
95. Wu X, Burgess SM. Integration target site selection for retroviruses and transposable elements. Cell Mol Life Sci 61:2588-2596.
96. Dav UP, Jenkins NA, Copeland NG. 2004. Gene therapy insertional mutagenesis insights. Science 303:333.
97. Kaiser J. 2004. Side effects sideline hemophilia trial. Science 304: 1423-1424.
98. Gao G, Lebherz C, Weiner DJ, Grant R, Calcedo R, et al. 2004. Erythropoietin gene therapy leads to autoimmune anemia in macaques. Blood 103:3300-3302.
99. Chenuaud P, Larcher T, Rabinowitz JE, Provost N, Cherel Y, et al. 2004. Autoimmune anemia in macaques following erythropoietin gene therapy. Blood 103:3303-3304.
100. Rivera VM, Gao G, Grant RL, Schnell MA, Zoltick PW, et al. 2005. Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer. Blood 105:1424-1430.