Targeted vectors for gene therapy

FASEB Journal

Volumn 9, Issue 2, 1995, Pages 190-199

  Miller, Nicholas ^a,b   Vile, Richard ^a  

^a ST THOMAS HOSPITAL   (United Kingdom)

^b GLAXO INST FOR MOLECULAR BIOLOGY   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

ASIALOGLYCOPROTEIN RECEPTOR; ASIALOOROSOMUCOID; COMPLEMENTARY DNA; EPIDERMAL GROWTH FACTOR; INSULIN; LECTIN; LIGAND; LIPOSOME; TRANSFERRIN; VIRUS ENVELOPE PROTEIN; VIRUS RECEPTOR;

ADENOVIRUS; ANTINEOPLASTIC ACTIVITY; ANTIVIRAL ACTIVITY; CLONING VECTOR; CONJUGATION; ECOTROPIC VIRUS; GENE TARGETING; GENE THERAPY; GENETIC ENGINEERING; GENETIC TRANSCRIPTION; HUMAN; LONG TERMINAL REPEAT; NONHUMAN; PRIORITY JOURNAL; RECOMBINANT GENE; RETICULOENDOTHELIAL SYSTEM; RETROVIRUS; REVIEW; VIRUS GENOME; VIRUS INFECTION;

ADENOVIRIDAE; UNIDENTIFIED RETROVIRUS;

EID: 0028946357     PISSN: 08926638     EISSN: None     Source Type: Journal    
DOI: 10.1096/fasebj.9.2.7781922     Document Type: Review

References (71)

1. Weiss, R. A. (1993) Cellular receptors and viral glycoproteins involved in retrovirus entry. In The Retroviridae (Levy, J. A., ed) pp. 1-108, Plenum, New York
2. Dalgleish, A. G., Beverley, P. C. L., Clapham, P. R., Crawford, D. H., Greaves, M. F., and Weiss, R. A. (1984) The CD4 (T4) antigen is an essential component of the receptor for the AIDS retrovirus. Nature312, 763
3. Poznansky, M., Lever, A., Bergeron, L., Haseltine, W., and Sodroski, J. (1991) Gene transfer into human lymphocytes by a defective human immunodeficiency virus type 1 vector. J. Virol.65, 532-536
4. Toggas, S. M., Masliah, F., Rockenstein, E. M., Rall, G. F., Abraham, C. R., and Mucke, L. (1994) Central nervous system damage produced by expression of the HIV-1 coat protein gp120 in transgenic mice. Nature 367, 188-193
5. Miller, A. D. (1992) Retroviral vectors. Curr. Top. Microbiol. Immunol.158, 1-24
6. Landau, N. R., Page, K. A., and Littman, D. R. (1991) Pseudotyping with human T-cell leukemia virus type I broadens the human immunodeficiency virus host range. J. Virol. 65, 162-169
7. Takeuchi, Y., Simpson, G., Vile, R. G., Weiss, R. A., and Collins, M. K. L. (1992) Retroviral pseudotypes produced by rescue of a Moloney murine leukemia virus vector by C-type, but not D-type, retroviruses. Virology186, 792-794
8. Vile, R. G., Schulz, T. F., Danos, O. F., Collins, M. K. L., and Weiss, R. A. (1991) A murine cell line producing HTLV-I pseudotype virions carrying a selectable marker gene. Virology 180, 420-424
9. Zhu, Z., Chen, S. S. L., and Huang, A. S. (1990) Phenotypic mixing between human immunodeficiency virus and vesicular stomatitis virus or herpes simplex virus. J. AIDS3, 215-219
10. Calafat, J., Janssen, H., Demant, P., Hilgers, J., and Zavada, J. (1983) Specific selection of host cell glycoproteins during assembly of murine leukaemia virus and vesicular stomatitis virus. J. Gen. Virol. 64, 1241-1253
11. Young, J. A. T., Bates, P., Willert, K., and Varmus, H. E. (1990) Efficient incorporation of human CD4 protein into avian leukosis virus particles. Science250, 1421-1423
12. st. J. Virol. 66, 4632-4638
13. Battini, J.-L., Heard, J. M., and Danos, O. (1992) Receptor choice determinants in the envelope glycoproteins of amphotropic, xenotropic and polytropic murine leukemia viruses. J. Virol. 66, 1468-1475
14. Morgan, R. A., Nussbaum, O., Muenchau, D. D., Shu, L., Couture, L., and French Anderson, W. (1993) Analysis of the functional and host range-determining regions of the murine ecotrupic and amphotropic retrovirus envelope proteins. J. Virol. 67, 4712-4721
15. Valsesia-Wittman, S., Drynda, A., Deleage, G., Aumailley, M., Heard, J.-M., Danos, O., Verdier, G., and Cosset, F.-L. (1994) Modifications in the binding domain of avian retrovirus envelope protein to redirect the host range of retroviral vectors. J. Virol.68, 4609-4619
16. Dong, J., Roth, M. G., and Hunter, E. (1992) A chimeric avian retrovirus containing the influenza virus hemagglutinin gene has an expanded host range. J. Virol. 66, 7374-7382
17. Brown, K. E., Anderson, S. M., and Young, N. S. (1993) Erythrocyte P antigen: cellular receptor for B19 parvovirus. Science262, 114-117
18. Rogers, G. N., Paulson, J. C., Daniels, R. S., Skehel, J. J., Wilson, I. A., and Wiley, D. C. (1983) Single amino acid substitutions in influenza haemagglutinin change receptor-binding specificity. Nature 304, 76-78
19. Etienne-Julan, M., Roux, P., Carillo, S., Jeanteur, P., and Piechaczyk, M. (2222) The efficiency of cell targeting by recombinant retroviruses depends on the nature of the receptor and the composition of the artificial cell-virus linker. J. Gen. Virol. 73, 3251-3255
20. Russell, S. J., Hawkins, R. E., and Winter, G. (1993) Retroviral vectors displaying functional antibody fragments. NAR21, 1081-1085
21. Neda, H., Wu, C. H., and Wu, G. Y. (1991) Chemical modification of an ecotropic murine leukemia virus results in redirection of its target cell specificity. J. Biol. Chem. 266, 14143-14146
22. Goud, B., Legrain, P., and Buttin, G. (1988) Antibody-mediated binding of a murine ecotropic Moloney retroviral vector to human cells allows internalisation but not the establishment of the proviral state. Virology163, 251-254
23. Roux, P., Jeanteur, P., and Piechaczyk, M. (1989) A versatile and potentially general approach to the targeting of specific cell types by retroviruses: application to the infection of human cells by means of major histocompatibility complex class I and class II antigens by mouse ecotropic murine leukemia virus-derived viruses. Proc Natl. Acad. Sci. USA 86, 9079-9083
24. 5 promote adenovirus internalisation but not virus attachment. Cell73, 309-319
25. Stratford-Perricaudet, L. D., Makeh, I., Perricaudet, M., and Briand, P. (1992) Widespread long-term gene transfer to mouse skeletal muscles and heart. J. Clin. Invest. 90, 626-6330
26. Wu, G. Y., Zhan, P., Sze, L. L., Rosenberg, A. R., and Wu, C. H. (1994) Incorporation of adenovirus into a ligand-based DNA carrier system results in retention of original receptor specificity and enhances targeted gene expression J. Biol. Chem.269, 11542-11546
27. Mouiller, P., Friedlander, G , Calise, D., Ronco, P., Perricaudet, M., and Ferry, N. (1994) Adenoviral-mediated gene transfer to renal tubular cells in vivo. Kidney Int. 45, 1220-1225
28. Allen, T. M. (1994) Long-circulating (sterically stabilised) liposomes for targeted drug delivery. TIPS15, 215-220
29. Nabel, E. G., Plautz, G., and Nabel, G. J. (1990) Site-specific gene expression in vivo by direct gene transfer into the arterial wall. Science 249, 1285-1288
30. Mizuno, M., Yoshida, J., Sugita, K., Inoue, I., Seo, H., Hayashi, Y., Koshizaka, T., and Yagi, K. (1990) Growth inhibition of glioma cells transfected with the human β-interferon gene by liposomes coupled with a monoclonal antibody. Cancer Res.50, 7826-7829
31. Stavridis, J. C., Deliconstantinos, G., Psallidopoulos, M. C., Armenakas, N. A., Hadjiminas, D. J., and Hadliminas, J. (1986) Construction of transferrin-coated liposomes for in vivo transport of exogenous DNA to bone marrow erythroblasts in rabbits. Exp. Cell Res. 164, 568-572
32. Walther, F. J., David-Cu, R., Supnet, M. C., Longo, M. L., Fan, B. R., and Bruni, R. (1993) Uptake of antioxidants in surfactant liposomes is enhanced by SP-A. Am. J. Physiol.265, L330-L339
33. Morishita, R., Gibbons, G. H., Kaneda, Y., Ogihara, T., and Dzau, V. J. (1993) Novel in vitro gene transfer method for study of local modulators in vascular smooth muscle cells. Hypertension 21, 894-899
34. Bagai, S., and Sarkar, D. P. (1993) Targeted delivery of hygromycin B using reconstituted Sendai viral envelopes lacking haemagglutinin-neuraminidase. FEBS Lett.326, 183-188
35. Schreier, H., Gonzalez-Rothi, R. J., and Stecenko, A. A. (1993) Pulmonary delivery of liposomes. J. Contr. Release 24, 209-223
36. Zhu, N., Liggitt, D., Liu, Y., and Debs, R. (1993) Systemic gene expression after intravenous DNA delivery into adult mice. Science261, 209-211
37. Michael, S. I., and Curiel, D. T. (1994) Strategies to achieve targeted gene delivery via the receptor-mediated endocytosis pathway. Gene Ther. 1, 223-232
38. Ferkol, T., Lindberg, G. L., Chen, J., Perales, J. C., Crawford, D. R., Ratnoff, O. D., and Hanson, R. W. (1993) Regulation of the phosphoenolpyruvate carboxykinase/human factor IX gene introduced into the livers of adult rats by receptor-mediated gene transfer. FASEB J. 7, 1081-1091
39. Rosenkranz, A. A., Yachmenev, S. V., Jans, D. A., Serebryakova, N. V., Murav'ev, V. I., Peters, R., and Sobolev, A. S. (1992) Receptor-mediated endocytosis and nuclear transport of a transfecting DNA construct. Exp. Cell Res.199, 323-329
40. Chen, J., Gamou, S., Takayanagi, A., and Shimizu, N. (1994) A novel gene delivery system using EGF receptor-mediated endocytosis FEBS Lett. 338, 167-169
41. Batra, R. K., Wang-Johanning, F., Wagner, E., Garver, R. I., and Curiel, D. T. (1994) Receptor-mediated gene delivery employing lectin-binding specificity. Gene Ther.1, 255-260
42. Michael, S. I., Huang, C.-H., Romer, M. U., Wagner, E., Hu, P-C., and Curiel, D. T. (1993) Binding-incompetent adenovirus facilitates molecular conjugate-mediated gene transfer by the receptor-mediated endocytosis pathway. J. Biol. Chem. 268, 6866-6869
43. Lozier, J. N., Thompson, A. R., Hu, P.-C., Read, M., and Brinkhouse, K. M. (1994) Efficient transaction of primary cells in a canine hemophilia B model using adenovirus-polylysine-DNA conjugates. Hum Gene Ther.5, 313-322
44. Wagner, E., Zenke, M., Cotten, M., Beug, H., and Birnstiel, M. L. (1990) Transferrin-polycation conjugates as carriers for DNA uptake into cells. Proc. Natl. Acad. Sci. USA 87, 3410-3414
45. Yang, Y., Nunes, F. A., Berencsi, K., Furth, E., Gonczol, E., and Wilson, J. M. (1994) Cellular immunity to viral antigens limits El-deleted adenoviruses for gene therapy. Proc. Natl. Acad. Sci. USA 91, 4407-4411
46. Hoeben, R. C., Migchielson, A. A. J., van der Jagt, R. C. M., van Ormondt, H., and van der Eb, A. J. (1991) Inactivation of the Moloney murine leukemia virus long terminal repeat in murine fibroblast cell lines is associated with methylation and is dependent on its chromosomal position. J. Virol.65, 904-912
47. Cox, G. A., Cole, N. M., Matsumura, K., Phelps, S. F., Hauschka, S. D., Campbell, K. P., Faulkner, J. A., and Chamberlain, J. S. (1993) Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity. Nature 364, 725-729
48. Maxwell, I. H., Glode, L. M., and Maxwell, F. (1991) Expression of the diphtheria toxin A-chain coding sequence under the control of promoters and enhancers from immunoglobulin genes as a means of directing toxicity to B-lymphoid cells. Cancer Res. 51, 4299-4304
49. Jahroudi, N., and Lynch, D. C. (1994) Endothelial-cell-specific regulation of von Willebrand factor gene expression. Mol. Cell. Biol. 14, 999-1008
50. Hatzoglou, M., Lamers, W., Bosch, F., Wynshaw-Boris, A., Clapp, D. W., and Hanson, R. W. (1990) Hepatic gene transfer in animals using retroviruses containing the promoter from the gene for phosphoenolpyruvate carboxykinase. J. Biol. Chem.265, 17285-17293
51. Vile, R. G., Miller, N., Chernajovsky, U., and Hart, I. R. (1994) A comparison of the properties of different retroviral vectors containing the murine tyrosinase promoter to achieve transcriptionally targeted expression of the HSVtk or IL-2 genes. Gene Ther. 1, 1-10
52. Friedman, J. M., Babiss, L. E., Clayton, D. F., and Darnell, J. E. (1986) Cellular promoters incorporated into the adenovirus genome: cell specificity of albumin and immunoglobulin expression. Mol. Cell. Biol. 6, 3791-3797
53. Brady, H. J. M., Miles, C. G., Pennington, D. J., and Dzierzak, E. A. (1994) Specific ablation of human immunodeficiency virus Tat-expressing cells. Proc. Natl. Acad. Sci. USA91, 365-369
54. Culver, K. W., Ram, Z., Wallbridge, S., Ishii, H., Oldfield, E. H., and Blaese, R. M. (1992) In vivo gene transfer with retroviral vector-producer cells for treatment of experimental brain tumours. Science 256, 1550-1552
55. Miyao, Y., Shimizu, K., Moriuchi, S., Yamada, M., Nakahira, K., Nakajima, K., Nakao, J., Kuriyama, S., Tsujii, T., Mikoshiba, K., Hayakawa, T., and Ikenaka, K. (1993) Selective expression of foreign genes in glioma cells: use of the mouse myelin basic gene promoter to direct toxic gene expression. J. Neurosci. Res.36, 472-479
56. Huber, B. E., Richards, C. A., and Krenitsky, T. A. (1991) Retroviral-mediated gene therapy for the treatment of hepatocellular carcinoma: an innovative approach for cancer therapy. Proc. Natl. Acad. Sci. USA 88, 8039-8043
57. Vile, R. G., and Hart, I. R. (1993) Use of tissue-specific expression of the herpes simplex virus thymidine kinase gene to inhibit growth of established murine melanomas following direct intra-tumoral injection of DNA. Cancer Res.53, 3860-3864
58. Harris, J. D., Gutierrez, A. A., Hurst, H. C., Sikora, K., and Lemoine, N. R. (1994) Gene therapy for cancer using tumour-specific prodrug activation. Gene Ther. 1, 170-175
59. Mayor, H. D. (1993) Defective parvoviruses may be good for your health. Prog. Med. Virol. 40, 193-205
60. Guetta, E., Graziani, Y., and Tal, J. (1986) Suppression of Ehrlich ascites tumors in mice by minute virus of mice. J. Natl. Cancer Inst.76, 1177-1180
61. Dupont, F., Tenenbaum, L., Guo, L.-P., Spegelaere, P., Zeicher, M., and Rommelaere, J. (1994) Use of an autonomous parvovirus vector for selective transfer of a foreign gene into transformed human cells of different origin and its expression therein. J. Virol. 68, 1397-1406
62. Huxley, C. (1994) Mammalian artificial chromosomes: a new tool for gene therapy. Gene Ther.1, 7-12
63. Arbones, M. L., Austin, H. A., Capon, D. J., and Greenburg, G. (1994) Gene targeting in normal somatic cells: inactivation of the interferon-γ receptor in myoblasts. Nature Genet. 6, 90-97
64. Shelling, A. N., and Smith, M. G. (1994) Targeted integration of transfected and infected adeno-associated virus vectors containing the neomycin resistance gene. Gene Ther.1, 165-169
65. Samulski, R. J. (1993) Adeno-associated virus: integration at a specific chromosomal locus. Curr. Opin. Genet. Dev. 3, 74-80
66. Chiorini, J. A., Weitzman, M. D., Owens, R. A., Urcelay, E., Safer, B., and Kotin, R. M. (1994) Biologically active Rep proteins of adeno-associated virus type 2 produced as fusion proteins in Escherichia coli J. Virol.68, 797-804
67. Flotte, T. R., Afione, S. A., Conrad, C., McGrath, S. A., Solow, R., Oka, H., Zeitlin, P. L., Guggino, W. B., and Carter, B. J. (1993) Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc. Natl. Acad. Sci. USA 90, 10613-10617
68. McDonald, J. F. (1993) Evolution and consequences of transposable elements. Curr. Opin. Genet. Dev.3, 855-864
69. Burns, N. R., Saibil, H. R., White, N. S., Pardon, J. F., Timmins, P. A., Richardson, S. M., Richards, B. M., Adams, S. E., Kingsman, S. M., and Kingsman, A. J. (1992) Symmetry, flexibility and permeability in the structure of yeast retrotransposon virus-like particles. EMBO J. 11, 1155-1164
70. Chakraborty, A. K., Zink, M. A., Boman, B. M., and Hodgson, C. P. (1993) Synthetic retrotransposon vectors for gene therapy. FASEB J. 7, 971-977
71. Voytas, D. F., and Bocke, J. D. (1993) Yeast retrotransposons and rRNAs. Trends Genet 9, 421-427