Gene therapy: The first decade

Trends in Biotechnology

Volumn 18, Issue 3, 2000, Pages 119-128

  Mountain, Andrew ^a  

^a KEELE UNIVERSITY   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ELECTROPORATION; EXPRESSION VECTOR; GENE EXPRESSION; GENE TECHNOLOGY; GENE THERAPY; GENE TRANSFER; HUMAN; MEDICAL GENETICS; NONHUMAN; PRIORITY JOURNAL; REVIEW;

ADENOVIRIDAE; CLINICAL TRIALS; DEPENDOVIRUS; GENE THERAPY; GENE TRANSFER TECHNIQUES; HUMANS; RETROVIRIDAE;

EID: 0034159940     PISSN: 01677799     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0167-7799(99)01416-X     Document Type: Review

References (89)

1. 1 Blaese M.et al. Vectors in cancer therapy: how will they deliver? Cancer Gene Ther. 2:1995;291-297.
2. 2 Wadman M. Germline gene therapy 'must be spared excessive regulation'. Nature. 392:1998;317.
3. 3 Robbins P.D.et al. Viral vectors for gene therapy. Trends Biotechnol. 16:1998;35-40.
4. 4 Carter B.J. Viral vectors for gene therapy. Mountain A.et al. Biotechnology (Vol. 5a, 2nd edn). 1999;395-426 Wiley-VCH.
5. 5 Robinson H.L.et al. Neutralizing antibody-independent containment of immunodeficiency virus challenges by DNA priming and recombinant pox virus booster immunisations. Nat. Med. 5:1999;526-534.
6. 6 Sanda M.G.et al. Recombinant vaccinia-PSA (PROSTVAC) can induce a prostate-specific immune response in androgen-modulated human prostate cancer. Urology. 53:1999;260-266.
7. 7 Krisky D.M.et al. Deletion of multiple immediate-early genes from herpes simplex virus reduces cytotoxicity and permits long-term gene expression in neurons. Gene Ther. 5:1998;1593-1603.
8. 8 Weir N. Non-viral vectors for gene therapy. Mountain A.et al. Biotechnology (Vol. 5a, 2nd edn). 1999;427-442 Wiley-VCH.
9. 9 Wolff J.A., Trubetskoy V.S. The Cambrian era of non-viral gene delivery. Nat. Biotechnol. 16:1998;421-422.
10. 165 following intramuscular gene transfer promotescollateral vessel development in patients with critical limb ischemia. Circulation. 97:1998;1114-1123.
11. 165 as sole therapy for myocardial ischemia. Circulation. 98:1998;2800-2804.
12. 12 Wadman M. Hyping results 'could damage' gene therapy. Nature. 378:1995;655.
13. 13 Searle P.F., Mautner V. Adenoviral vectors: not to be sneezed at. Gene Ther. 5:1998;725-727.
14. 14 Bilbao G.et al. Improving adenoviral vectors for gene therapy. Tumor Targeting. 3:1998;59-79.
15. 15 Parr M.J. Tumor-selective transgene expression in vivo mediated by an E2F-responsive adenoviral vector. Nat. Med. 3:1997;1145-1149.
16. 16 Schiedner G.et al. Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity. Nat. Genet. 18:1998;180-183.
17. 17 Kaplan J.M.et al. Humoral and cellular immune responses of nonhuman primates to long-term repeated lung exposure to Ad2/CFTR-2. Gene Ther. 3:1996;117-127.
18. 18 Chirmule N.et al. Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther. 6:1999;1574-1583.
19. 19 Swisher S.G.et al. Adenovirus-mediated p53 gene transfer in advanced non-small-cell lung cancer. J. Natl. Cancer Inst. 91:1999;763-771.
20. 20 Ilan Y.et al. Oral tolerization to adenoviral antigens permits long-term gene expression using recombinant adenoviral vectors. J. Clin. Invest. 99:1997;1098-1106.
21. 21 Beer S.J.et al. Poly (lactic-glycolic) acid copolymer encapsulation of recombinant adenovirus reduces immunogenicity in vivo. Gene Ther. 5:1998;740-746.
22. 22 Chillon M.et al. Adenovirus complexed with polyethylene glycol and cationic lipid is shielded from neutralizing antibodies in vitro. Gene Ther. 5:1998;995-1002.
23. 23 Stein C.S.et al. Effects of macrophage depletion and anti-CD40 ligand on transgene expression and redosing with recombinant adenovirus. Gene Ther. 5:1998;431-439.
24. 24 Yang Y.et al. Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses. J. Virol. 69:1995;2004-2015.
25. 25 Jooss K.et al. Blunting of immune responses to adenoviral vectors in mouse liver and lung with CTLA4Ig. Gene Ther. 5:1998;309-319.
26. 26 Benihoud K.et al. Efficient repeated adenovirus-mediated gene transfer in mice lacking both tumour necrosis factor alpha and lymphotoxin alpha. J. Virol. 72:1998;9514-9525.
27. 27 Amalfitano A.et al. Production and characterization of improved adenovirus vectors with the E1, E2b, and E3 genes deleted. J. Virol. 72:1998;926-933.
28. 28 Fallaux F.J.et al. New helper cells and matched early region 1-deleted adenovirus vectors prevent generation of replication-competent adenovirus. Hum. Gene Ther. 9:1998;1909-1917.
29. 29 Kuryama S.et al. Expression of a retrovirally transduced gene under control of an internal housekeeping gene promoter does not persist due to methylation and is restored partially by 5-azacytidine treatment. Gene Ther. 5:1998;1299-1305.
30. 30 Miller N., Whelan J. Progress in transcriptionally targeted and regulatable vectors for genetic therapy. Hum. Gene Ther. 8:1997;803-815.
31. 31 Riddell S.et al. T-cell mediated rejection of gene-modified HIV-specific cytotoxic T lymphocytes in HIV-infected patients. Nat. Med. 2:1996;216-223.
32. 32 Ory D.S.et al. A stable human-derived packaging cell line for production of high titer retrovirus/vesicular stomatitis virus G pseudotypes. Proc. Natl. Acad. Sci. U. S. A. 93:1996;11400-11406.
33. 33 Zufferey R.et al. Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nat. Biotechnol. 15:1997;871-875.
34. + cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science. 283:1999;682-686.
35. 35 Balague C.et al. Adeno-associated virus Rep78 protein and terminal repeats enhance integration of DNA sequences into the cellular genome. J. Virol. 71:1997;3299-3306.
36. 36 Donahue B.A.et al. Selective uptake and sustained expression of AAV vectors following subcutaneous delivery. J. Gene Med. 1:1999;31-42.
37. 37 Linden R.M., Woo S.L. AAVant-garde gene therapy. Nat. Med. 5:1999;21-22.
38. 38 Conrad C.K.et al. Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung. Gene Ther. 3:1996;658-668.
39. 39 Wagner J.A. Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus. Lancet. 351:1998;1702-1703.
40. 40 Xiao W.et al. Gene therapy vectors based on adeno-associated virus type 1. J. Virol. 73:1999;3994-4003.
41. 2 antibody. Nat. Biotechnol. 17:1999;181-186.
42. 42 During M.J.et al. Peroral gene therapy of lactose intolerance using an adeno-associated virus. Nat. Med. 4:1999;1131-1135.
43. 43 Ferrari F.K.et al. New developments in the generation of Ad-free, high-titer rAAV gene therapy vectors. Nat. Med. 3:1997;1295-1298.
44. 44 Matsushita T.et al. Adeno-associated virus vectors can be efficiently produced without helper virus. Gene Ther. 5:1998;938-945.
45. 45 Wolff J.A.et al. Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle. Hum. Mol. Genet. 1:1992;363-369.
46. 46 Hengge U.et al. Cytokine gene expression in epidermis with biological effects following injection of naked DNA. Nat. Genet. 10:1995;161-166.
47. 47 Wang R.et al. Induction of antigen-specific cytotoxic T lymphocytes in humans by a malaria DNA vaccine. Science. 282:1998;476-480.
48. 48 Krieg A.M.J. Direct immunologic activities of CpG DNA and implications for gene therapy. J. Gene Med. 1:1999;56-63.
49. 49 Brigham K.L.et al. In vivo transfection of murine lungs with a functioning prokaryotic gene using a liposome vehicle. Am. J. Med. Sci. 298:1989;278-281.
50. 50 Alton E.W.et al. Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice. Nat. Genet. 5:1993;135-142.
51. 51 Alton E.W.et al. Towards gene therapy for cystic fibrosis: a clinical progress report. Gene Ther. 5:1998;291-292.
52. 52 Davies J.C.et al. Prospects for gene therapy for cystic fibrosis. Mol. Med. Today. 4:1998;292-299.
53. 53 Li S., Huang L. In vivo gene transfer via intravenous administration of cationic lipid-protamine-DNA (LPD) complexes. Gene Ther. 4:1997;891-900.
54. 54 Xing X.et al. Safety study and characterization of E1A-liposome complex gene-delivery protocol in an ovarian cancer model. Gene Ther. 5:1998;1538-1544.
55. 55 Stopeck A.T.et al. Phase I study of direct gene transfer of an allogeneic histocompatibility antigen, HLA-B7, in patients with metastatic melanoma. J. Clin. Oncol. 15:1997;341-349.
56. 56 Wagner E.et al. Transferrin-polycation conjugates as carriers for DNA uptake into cells. Proc. Natl. Acad. Sci. U. S. A. 87:1990;3410-3414.
57. 57 Gottschalk S.et al. A novel DNA-peptide complex for efficient gene transfer and expression in mammalian cells. Gene Ther. 3:1996;448-457.
58. 58 Philips, R.et al. (1998) International Patent Application PCT/GB98/00424.
59. 59 Boussif O.et al. Optimized galenics improve in vitro gene transfer by cationic molecules up to 1000-fold. Gene Ther. 3:1996;1074-1080.
60. 60 Goula D.et al. Polyethyleneimine-based intravenous delivery of transgenes to mouse lung. Gene Ther. 5:1998;1291-1295.
61. 61 Kircheis R.et al. Coupling of cell-binding ligands to polyethyleneimine for targeted gene delivery. Gene Ther. 4:1997;409-418.
62. 62 Condon C.et al. DNA-based immunisation by in vivo transfection of dendritic cells. Nat. Med. 2:1996;1122-1128.
63. 63 Mathei C.et al. Hepatitis B vaccine administration: comparison between jet-gun and syringe and needle. Vaccine. 15:1997;402-404.
64. 64 Tacket C.O.et al. Phase 1 safety and immune response studies of a DNA vaccine encoding hepatitis B surface antigen delivered by a gene delivery device. Vaccine. 17:1999;2826-2829.
65. 65 Nishi T.et al. Treatment of cancer using pulsed electric field in combination with chemotherapeutic agents or genes. Hum. Cell. 10:1997;81-86.
66. 66 Zhang L.et al. Depth-targeted efficient gene delivery and expression in the skin by pulsed electric fields: an approach to gene therapy of skin aging and other diseases. Biochem. Biophys. Res. Commun. 220:1996;633-636.
67. 67 Keller G., Snodgrass R. Human embryonic stem cells: the future is now. Nat. Med. 5:1999;151-152.
68. 68 Fraser P., Grosveld F. Locus control regions, chromatin activation and transcription. Curr. Opin. Genet. Dev. 9:1998;152-157.
69. 69 Webster K.A. Molecular switches for regulating therapeutic genes. Gene Ther. 6:1999;951-953.
70. 70 Ye X.et al. Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer. Science. 283:1999;88-91.
71. 71 Aiyar A.et al. The plasmid replicon of EBV consists of multiple cis-acting elements that facilitate DNA synthesis by the cell and a viral maintenance element. EMBO J. 17:1998;6394-6403.
72. 72 Cooper M.J.et al. Safety-modified episomal vectors for human gene therapy. Proc. Natl. Acad. Sci. U. S. A. 94:1997;6450-6455.
73. 73 Willard H.F. Human artificial chromosomes coming into focus. Nat. Biotechnol. 16:1998;415-417.
74. 74 Surosky R.T.et al. Adeno-associated virus Rep proteins target DNA sequences to a unique locus in the human genome. J. Virol. 71:1997;7951-7959.
75. 75 Feng M.et al. Stable in vivo gene transduction via a novel adenoviral/retroviral chimeric vector. Nat. Biotechnol. 15:1997;866-870.
76. 76 Perricaudet, M. et al. (1999) Abstracts of American Society of Gene Therapy 2nd Annual Meeting, Washington, DC, 9-13 June, 1999.
77. 77 Russell D.W., Hirata R.K. Human gene targeting by viral vectors. Nat. Genet. 18:1998;325-330.
78. 78 Kmiec E.B. Targeted gene repair. Gene Ther. 6:1999;1-3.
79. +-enriched cell population using a chimeric RNA/DNA oligonucleotide. J. Mol. Med. 75:1997;829-835.
80. 80 Kren B.T.et al. In vivo site-directed mutagenesis of the factor IX gene by chimeric RNA/DNA oligonucleotides. Nat. Med. 4:1998;285-290.
81. 81 Billings P.R. In utero gene therapy - the case against. Nat. Med. 5:1999;255-256.
82. 82 Schneider H., Coutelle C. In utero gene therapy - the case for. Nat. Med. 5:1999;256-257.
83. 83 Walsh C.E. Fetal gene therapy. Gene Ther. 6:1999;1200-1201.
84. 84 Wadman M. Gene therapy pushes on, despite doubts. Nature. 397:1999;94.
85. 85 Wadman M. Congress may block stem cell research. Nature. 397:1999;639.
86. 86 Updated list of clinical protocols (1999) Hum. Gene Ther. 10, 1043-1092.
87. 87 Abstracts of American Society of Gene Therapy 2nd Annual Meeting, Washington, DC, 9-13 June, 1999.
88. 88 Kirn D.et al. ONYX-015: clinical data are encouraging. Nat. Med. 4:1998;1341-1342.
89. 89 Ram Z.et al. Therapy of malignant brain tumours by intratumoral implantation of retroviral vector-producing cells. Nat. Med. 3:1997;1354-1361.