Fundamentals and future prospects of gene therapy

Drugs of the Future

Volumn 22, Issue 9, 1997, Pages 995-1003

  Singh, M ^a   Parikh, V ^a   Sharma, A ^a  

^a PUNJABI UNIVERSITY   (India)

Author keywords

[No Author keywords available]

Indexed keywords

ACQUIRED IMMUNE DEFICIENCY SYNDROME; CARDIOVASCULAR DISEASE; EXPRESSION VECTOR; FOOD AND DRUG ADMINISTRATION; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENETIC DISORDER; HEMATOPOIETIC CELL; HUMAN; LIVER DISEASE; LUNG DISEASE; MALIGNANT NEOPLASTIC DISEASE; MEDICAL ETHICS; NEUROLOGIC DISEASE; NONHUMAN; REVIEW;

EID: 0030844111     PISSN: 03778282     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (95)

1. Verma, I. Gene therapy. Sei Am 1990, 263: 68-84.
2. Mulligan, R.C. The basic science of gene therapy. Science 1993, 260: 926-32.
3. Recombinant Advisory Committee. Points to consider in he design and submission of human somatic eel I gene therapy protocols. Recomb DNA Tech Bull 1986, 9:221-5.
4. Hammer, R.E., Palmiter, R.D., Brinster, R.L. Partial correction of a murine hereditary growth disorder by germ line incorporation of a new gene. Nature 1984, 311:65-7.
5. Costantini, F., Chad, K., Magram, J. Correction of murine β-thalassemia by gene transfer into the germ line. Science 1986, 233: 1192-4.
6. Jones, S.N., Gropme, M., Munir, M.I. et al. Ectopic correction of ornithine carbamyl transferase deficiency in sparse fur mice. J Biol Chem 1990, 265:14684-6.
7. Kyle, J.W., Birkenmier, E.T., Gwynn, B. et al. Correction of murine mucopolysaccharidosis VII by a human ß-glucuronidase transgene. Proc Natl Acad Sei USA 1990, 87: 3914-7.
8. Harbers, K., Kuehn, M., Delius, H., Jenisch, R. Insertion of retrovirus into the first intron ofal(1) collagen gene leads to embryonic lethal mutation in mice. Proc Natl Acad Sei USA 1984,81:1504-7.
9. Eglitis, M.A., Kantoff, P.W., Gilboa, E., Andersen, W.F. Gene expression in mice after high efficiency retroviral mediated gene transfer. Science 1985,230:1395-7.
10. Magli, M.C., Dick, J.E., Huszar, D., Bernstein, A., Philips, R.A. Modulation of gene expression in multiple hematopoietic cell lineages following retroviral vector gene transfer. Proc Natl Acad Sci USA 1987, 84:789-93.
11. Hock, R.A., Miller, A.D. Retrovirus mediated transfer and expression of drug resistance genes in human hematopoietic cells in vivo. Nature 1986, 320: 275-7.
12. Williams, D.A., Orkin, S.H., Mulligan, R.C. Retrovirus mediated transfer of human adenosine deaminasegene sequences into cells in culture and into murine hematopoietic cells in vivo. Proc Natl Acad Sei 1986, 83: 2566-9.
13. Seiden, R.F., Skoskiewics, M.J., Howie, K.B. et al. Implantation of genetically engineered fibroblasts into mice: Implications for gene therapy. Science 1987, 236: 714-8.
14. Miyanohara, A., Sharkey, M.F., Witztum, J.L. et al. Efficient expression of retroviral vector transduced human low density lipoprotein (LDL) receptor in LDL receptor deficient rabbit fibroblasts in vitro. Proc Natl Acad Sei USA 1988, 85: 6538-42.
15. Palmer, T.D., Rasman, G.J., Osborne, W.R.A., Miller, A.D. Genetically modified skin fibroblasts persist long after transplantation but gradually activated introduced genes. Proc Natl Acad Sei USA 1991, 88:1330-3.
16. Wolffe, J.A., Yee, J.K., Skell, H.F. etal. Expression of retrovirally transduced genes in primary cultures of adult rat hepatocytes. Proc Natl Acad Sei USA 1987, 84: 3344-6.
17. Wilson, J.M., Jefferson, D.M., Chowdhury, J.R. et al. Retrovirus mediated transduction of adult hepatocytes. Proc Natl Acad Sci USA 1988, 85:3014-7.
18. Zweibel, J.A., Freeman, S.M., Kantoff, p.w. et al. High level recombinant gene expression in rabbit endothelial cells transduced retroviral vectors. Science 1989, 243: 220-2.
19. Morgan, J.R., Barrandon, Y., Green, H., Mulligan, R.C. Expression of an exogenous growth hormone gene by transplantable human epidermal cells. Science 1987,237:1476-9.
20. Fenjves, E.S., Gordon, D.A., Pershing, U.K., Williams, D.L. Systemic distribution of apolipoprotein E secreted by grafts of epidermal keratinocytes: Implication for epidermal function and gene therapy. Proc Natl Acad Sei USA 1989, 85: 8803-7.
21. Gilboa, E., Eglitis, M.A., Kantoff, P.N., Anderson, W.F. Transfer and expression of clonedgenes using retroviral vectors. Biotechniques 1986, 4:504-12.
22. Eglitis, M.A., Anderson, W.F. Retroviral vectors for introduction of genes into mammalian cells. Biotechniques 1988, 6: 608-14.
23. McLachlin, J.R., Cornetta, K-. Eglitis, M.A., Anderson, W.F. Retroviral mediated gene transfer. Prog Nucleic Acid Res Mol Biol 1990,38:91-135.
24. Varmus, H. Retroviruses. Science 1988, 240:1427-35.
25. Berkner, K.L. Development ofadenovirus vector forthe expression of heterologous genes. Biotechniques 1988, 6: 616-28.
26. Rosenfeld, M.A., Siegfried, W., Yoshimura, K. et al. Adenovirus mediated transfer of a recombinantai -antitrypsin gene to the lung epithelium in vivo. Science 1991, 259: 431-4.
27. Rosenfeld, M.A., Yoshimura, K., Trapnell, B.C. et al. In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium. Cell 1992, 68:143-55.
28. McLaughlin, S.K., Collis, P., Hermonat, P., Muzyczka, N. Adeno-associated virus general transduction vector: Analysis of proviral structures. J Virol 1988, 63:1963-73.
29. Graham, F.L., Van der Eb, A.J. A new technique for the assay of infectivity of human- adenovirus 5 ON A. Virology 1973, 52: 456-67.
30. Brooks, G. Gene therapy. Pharm J 1994, 252: 256-60.
31. Nabel, E.G., Plautz, G., Nabel, G.J. Site specific gene expression in vivo by direct gene transfer into arterial wall. Science 1990, 249:1285-8.
32. Wolffe, J.A., Malone, R.W., Williams, P. et al. Direct gene transfer into mouse muscle in vivo. Science 1990, 247:1465-8.
33. Nelson, D.L, Cox, M.M. Recombinant DNA technology. In: Principles of Biochemistry, 2nd Ed. Lehninger, A.L., Nelson, D.L., Cox, M.M. (Eds.). Worth Publishers: New York 1993,1005-7.
34. Wu, C.H., Wilson, J.M., Wu, G.Y. Targeting genes: Delivery and persistent expression of a foreign gene driven by mammalian regulatory elements in vivo. J Biol Chem 1989, 264:16985-7.
35. Zenke, M., Steinlein, P., Wagner, E. et al. Receptor mediated endocytosis oftransferrin-poly-cation conjugates: An efficient way to introduce DNA into hematopoietic cells. Proc Natl Acad Sei USA 1990,87:3655-9.
36. Curiel, D.T., Wagner, E., Cotton, M. et al. High efficiency gene transfer mediated by adenovirus coupled to DNA-polylysine complexes. Hum Gene Ther 1992, 3:147-154.
37. Bodine, D.M., Karlsson, S., Nienhuis, A.W. Combination of interleukin-3 and -6 preserves stem cell function in culture and enhances retrovirus mediated gene transfer into hematopoietic stem cells. Proc Natl Acad Sei USA 1989, 7: 8901-4.
38. Williams, D.E., Eisman, J., Baird, A. et al. Identification of a ligandforthe C-kit proto-oncogene. Cell 1990, 63: 167-70.
39. Zsebo, K.M., Wypych, J., McNiece, I.K. et al. Identification,purification and biological characterisation of hematopoietic stem cell factor from buffalo rat liver conditioned medium. Cell 1990, 63: 195-201.
40. Berardi, A.C., Anlai, W., Levine, J.D., Lopez, P., Scadden, D.T. Functional isolation and characterisation of human hematopoietic cells. Science 1995, 267:104-8.
41. Szilvassy, S.J., Eraser, C.C., Eaves, C.J. et al. Retrovirus mediated gene transfer to purified hematopoietic stem cells with long term lymphomyelopoietic repopulating ability. Proc Natl Acad Sci USA 1989, 86: 8798-802.
42. Spangrude, G.J., Heimfeld, S., Weissman, l.L. Purification and characterization of mouse hematopoietic stem cells. Science 1988, 241:58-62.
43. Van, D.R., Heimfeld, S., Spangrude, G.J., Weissman, l.L. Mouse hematopoietic stem cell antigen Sca-1 is a member of the Ly-6 antigen family. Proc Natl Acad Sei USA 1989, 86: 6434-7.
44. Ledlet, F.D., Grenett, H.E., McGinnis-Shelnutt, M., Woo, S.L.C. Retroviral mediatedgene transfer of human phenylalanine hydroxylase into NIH 3T3 and hepatoma cells. Proc Natl Acad Sei USA 1986,83:409-13.
45. Sorge, J., Kühl, W., West, C., Beutler, E. Complete correction of the enzymatic defect of type l Gaucher disease fibroblast by retroviral mediated gene transfer. Proc Natl Acad Sei USA 1987, 84: 906-9.
46. Ponder, K., Gupta, S., Leland, F. et al. Mouse hepatocytes migrate to liver parenchyma and function indefinitely after intrasplenic transplantation. Proc Natl Acad Sei USA 1991, 88:1217-21.
47. Demetriou, A.A., Whiting, J.F., Fedlman, D., Levenson, S. Replacement of liver function in rats by transplantation and microcarrier attached hepatocytes. Science 1986, 233:1190-2.
48. Demetriou, A.A., Levenson, S.M., Novikorf, P.M., Novikoff, A.B. Survival, organization and function of microcarrier attached hepatocytes transplanted in rats. Proc Natl Acad Sei USA 1986, 83: 7475-8.
49. Anderson, K.D., Thomson, U.A., DiPietro, J.M. et al. Gene expression in implanted rat hepatocytes following retroviral mediatedgene transfer. Somat Cell Mol Genet 1989,15:215-27.
50. Wu, G.Y., Wu, C.H. Receptor-mediated gene delivery and expression in vivo. J Biol Chem 1988, 263:14621-4.
51. Dickinson, P.D., Dorin, J.R., Porteous, D.J. Modelling cystic fibrosis in mouse. Mol Med Today 1995,1:140-8.
52. Barinaga, M. Gene therapy for clogged arteries passes test in pigs. Science 1994, 265: 738.
53. Chang, M.W., Barr, E., Seltzer, J. Cytostatic gene therapy for vascular proliferative disorders with a constitutively active form of the retinoblastoma gene product. Science 1995, 267: 518-22.
54. Leinwand, L.A., Leiden, J.M. Gene transfer into cardiac myocytes in vivo. Trends Cardiovasc Med 1991,1: 271-6.
55. St. Louis, D., Verma, I.M. An alternative approach to somatic cell gene therapy. Proc Natl Acad Sei USA 1988, 85: 3150-2.
56. Bubenik, J. Cytokine gene modified vaccines in the therapy of cancer. Pharmacol Ther 1996, 69:1-14.
57. Apscott, S.J., Miller, A.J., Oison, J.M. et al. Gene therapy of rat 9L gliosarcoma tumors by transduction with selectable gene does not require drug selection. Proc Natl Acad Sei USA 1994, 91: 8185-9.
58. Vile, R.G., Hart, I.R. Use of tissue gene to inhibit specific expression of the herpes simplex virus thymidine kinase growth of established murine melanomas following direct intratumoral injection ofDNA. Cancer Res 1993, 53: 3860-4.
59. Sorrentino, B.P., Brandt, S.J., Bodine, D. Selection of drug resistant bone marrow cells in vivo after retroviral transfer of human MDR1. Science 1992, 257: 99-103.
60. Fan, T.P.D., Jaggar, R., Bicknell, R. Controlling the vasculature: Angiogenesis, anti-angiogenesis and vascular targeting of gene therapy. Trends Pharmacol Sei 1995,16: 57-66.
61. Millauer, B., Shawver, L.K., Plate, K.H. et al. Glioblastoma growth inhibited in vivo by a dominant negative FIK-1 mutant. Nature 1994, 367:576-9.
62. Baltimore, D. Intracellular immunisation. Nature 1989, 335-395-6.
63. Chatterjee, S., Johnson, P.R., Wong, K.K. Dual target inhibition ofHIV-1 in vitro by means of an adeno-associated virus antisense vector. Science 1992, 258:1485-8.
64. Friedman, T. Progress toward human gene therapy. Science 1989,244:1275-81.
65. Acsadi, G., Dickson, G., Love, D.R. et al. Human dystrophin expression in mdr mice after intramuscular injection of DNA constructs. Science 1991, 352: 815-8.
66. During, M.J., Naegele, J.R., O'Malley, K.L., Geller, A.I. Long term behavioral recovery in parkinsonian rats by an HSV vector expressing tyrosine hydroxylase. Science 1994, 266:1399-403.
67. Icacson, l.. Geller, A.I., Naegele, J.R., O'Malley, K.L, During, M.J. Behavioral effects and gene delivery in a rat model of Parkinson's disease. Science 1995, 269: 856-7.
68. Millet, J. Catecholamines: From gene regulation to neuropsychiatrie disorders. Trends Pharmacol Sei 1996,17:129-35.
69. Mitsumoto, H., Ikeda, K., Klinskosz, B. et at. Arrest of motor neuron disease in Wobbler mice cotreated with CNTFandBDNF. Science 1994, 265: 1107-10.
70. Matsumoto, M., Nakagawa, T., Inoue, T. et al. Ataxia andepileptic seizures in mice lacking type I inositol 1,4,5-triphosphate receptor. Nature 1996, 379:168-71.
71. Kasid, A., Morecki, S., Aebersold, P. et al. Humangene transfer: Characterisation of human tumor infiltrating lymphocytes as vehicles for retroviral mediated gene transfer in man. Proc Natl Acad Sei USA 1990, 87: 473-7.
72. Rosenberg, S.A., Aebersold, P., Cornelia, K. et al. Gene transfer into humans - Immunotherapy of patients with advanced melanoma using tumor infiltrating lymphocytes modified by retroviral gene transduction. New Engl J Med 1990, 326: 370-8.
73. Anderson, W.F. Human gene therapy. Science 1992, 256: 808-13.
74. Blease, R.M., Culver, K.W., Miller, A.D. et al. T lymphocyte directed gene therapy for ADA SCID: Initial trial results after 4 years. Science 1995, 270: 475-80.
75. Bordignon, C., Notarangelo, L.D., Nobili, N. et al. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA immunodeficientpatients. Science 1995, 270: 470-4.
76. Meager, A., Griffith, E. Human somatic gene therapy. Trends BiotechnoM 994,13:108-13.
77. Watson, T. Gene therapy research is spread across many disciplines and institutes at NIH. Nature 1992, 359:188-9.
78. Thompson, J.D., Macejak, D., Couture, L, Stinchcomb, D.T. Piibozymes in gene therapy. Nat Med 1995,1: 277-9.
79. Rosenberg, S.A. The immunotherapy andgene therapy ofcancer. J Clin Oncol 1992, 10:180-99.
80. Crowley, N.H., Siegler, H.F. Possibilities of immunotherapy and gene therapy for malignant melanoma. Semin Surg Oncol 1993,9: 273-8.
81. Isobe, K.I., Zuo Hang, F., Nobuhiko, E., Izumi, N. Gene transfer of TNF receptor for treatment of cancer by TNF. Biochem Biophys Res Commun 1994, 203:1538-42.
82. Milano, C.A., Allen, L.F., Rockman, H.A. et al. Enhancedmyocardial function in transgenic mice overexpressing the ß2-adrenergic receptor. Science 1994, 264: 582-6.
83. Martin, J.B. Gene therapy and pharmacological treatment of inherited neurological disorders. Trends Biotechnol 1995, 13: 28-35.
84. Rosen, D.R., Siddiqui, T., Patterson, D. et al. Mutations in the Cu/Zn Superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis. Nature 1993, 362: 59-62.
85. Gurney, M.E., Pu, H., Chiu, A.Y. étal. Motor neuron degeneration in mice that express a human Cu, Zn Superoxide dismutase (nutation. Science 1994, 264:1772-5.
86. g6. Rommens, J.M., lannuzi, M.C., Kerem, B.S. et al. Identification of the cystic fibrosis gene. Science 1989, 245:1059-80.
87. Coutelle, C., Caplen, N., Hart, S. et al. Gene therapy for cystic fibrosis. Arch Dis Child 1993, 68: 437-43.
88. Dwarki, V.J., Belloni, P., Nijjar, T. et al. Gene therapy for hemophilia A: Production of therapeutic levels of human factor VIII in vivo in mice. Proc Natl Acad Sei USA 1995, 92:1023-7.
89. Kay, M.A., Rothenberg, S., Landen, C.M. et al. In vivogene therapy of hemophilia B: Sustained partial correction in factor IX deficient dogs. Science 1993, 262:117-9.
90. Koldka, T.M., Finegold, M., Moss, L, Woo, S.L.C. Gene therapy for diabetes mellitus in rats by hepatic expression of insulin. Proc Natl Acad Sei USA 1995, 92: 3293-7.
91. Ally, A.B., Hawley, T.S., McKali-Faienza, K.J. et al. Prevention of autoimmune disease by retroviral-mediated gene therapy. J Immunol 1995,155: 5404-8.
92. Ichiro, K., Suzuki, Y, Akabane, A. et al. Transgenic mice overexpressing human vasoactive intestinal peptide (VlP)gene in pancreatic beta cells: Evidence for improved glucose tolerance and enhanced insulin secretion by VIP and PHM-27 in vivo. J Biol Chem 1994,269:21223-8.
93. Temin, H.M. Safety consideration in somatic gene therapy of human disease with retroviral vector. Hum Gene Ther 1990, 1: 111-23.
94. Cornelia, K., Morgan, R.A., Anderson, W.F. Safety issues related to retroviral mediated gene transfer in humans. Hum Gene Ther 1991, 2: 5-14.
95. Walters, L. The ethics of human gene therapy. Nature 1986, 320:225-7.