Human gene therapy

Nature

Volumn 392, Issue 6679 SUPPL., 1998, Pages 25-30

  Anderson, W French ^a  

^a UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CLINICAL RESEARCH; EXPRESSION VECTOR; GENE THERAPY; HUMAN; MEDICAL ETHICS; MEDICAL GENETICS; PRIORITY JOURNAL; REVIEW; SIGNAL TRANSDUCTION;

CLINICAL PROTOCOLS; CLINICAL TRIALS; DNA VIRUSES; DRUG DELIVERY SYSTEMS; ETHICS, MEDICAL; GENE THERAPY; GENETIC VECTORS; HUMANS; RNA VIRUSES;

EID: 0032580348     PISSN: 00280836     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (56)

1. Blaese, R. M. et al. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science 270, 475-480 (1995).
2. Kay, M. A., Liu, D. & Hoogerbrugge, P. M. Gene therapy. Proc. Natl Acad. Sci. USA 94, 12744-12746 (1997).
3. Verma, I. M. & Somia, N. Gene therapy: promises, problems and prospects. Nature 389, 239-242 (1997).
4. Havenga, M., Hoogerbrugge, P., Valerio, D. & van Es, H. H. G. Retroviral stem cell gene therapy. Stem Cells 15, 162-179 (1997).
5. Dass, C. R. et al. Cationic liposomes and gene therapy for solid tumors. Drug Delivery 4, 151-165 (1997).
6. Miller, N. & Whelan, J. Progress in transcriptionally targeted and regulatable vectors for genetic therapy. Hum. Gene Ther. 8, 803-815 (1997).
7. Cosset, F.-L. & Russell, S. J. Targeting retrovirus entry. Gene Ther. 3, 946-956 (1996).
8. Cristiano, R. J. & Curiel, D. T. Strategies to accomplish gene delivery via the receptor-mediated endocytosis pathway. Cancer Gene Ther. 3, 49-57 (1996).
9. Brenner, M. Gene marking. Hum. Gene Ther. 7, 1927-1936 (1996).
10. Schnierle, B. S. & Groner, B. Retroviral targeted delivery. Gene Ther. 3, 1069-1073 (1996).
11. Anderson, W. F. Prospects for human gene therapy. Science 226, 401-409 (1984).
12. Orlic, D. et al. The level of mRNA encoding the amphotropic retrovirus receptor in mouse and human hematopoietic stem cells is low and correlates with the efficiency of retrovirus transduction. Proc. Natl Acad. Sci. USA 93, 11097-11102 (1996).
13. Salmons, B. & Gunzburg, W. H. Targeting of retroviral vectors for gene therapy. Hum. Gene Ther. 4, 129-141 (1993).
14. Kasahara, N. A., Dozy, A. M. & Kan, Y. W. Tissue-specific targeting of retroviral vectors through ligand- receptor interactions. Science 266, 1373-1376 (1994).
15. Fass, D., Harrison, S. C. & Kim, P. S. Retrovirus envelope domain at 1.7 A resolution. Nature Struct. Biol. 3, 465-469 (1996).
16. Fass, D. et al. Structure of a murine leukemia virus receptor-binding glycoprotein at 2.0 angstrom resolution. Science 277, 1662-1666 (1997).
17. Zhao, Y., Lee, S. & Anderson, W. F. Functional interactions between monomers of the retroviral envelope protein complex. J. Virol. 71, 6967-6972 (1997).
18. Zhao, Y. et al. Functional domains in retroviral transmembrane protein. J. Virol. (in the press).
19. Hanenberg, H. et al. Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells. Nature Med. 2, 876-882 (1996)
20. Hall, F. L. et al. Targeting retroviral vectors to vascular lesions by genetic engineering of the MoMLV gp70 envelope protein. Hum. Gene Ther. 8, 2183-2192 (1997).
21. Zufferey, R. et al. Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nature Biotechnol. 15, 871-875 (1997).
22. Kafri, T. et al. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nature Genet. 17, 314-317 (1997).
23. Russell, D. W. & Miller, A. D. Foamy virus vectors. J. Virol. 70, 217-222 (1996).
24. Donahue, R. E. et al. Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer. J. Exp. Med. 176, 1125-1135 (1992).
25. Anderson, W. F., McGarrity, G. J. & Moen, R. C. Report to the NIH Recombinant DNA Advisory Committee on murine replication-competent retrovirus (RCR) assays. Hum. Gene Ther. 4, 311-321 (1993).
26. Forestell, S. P. et al. Novel retroviral packaging cell lines: complementary tropisms and improved vector production for efficient gene transfer. Gene Ther. 4, 600-610 (1997).
27. Ali, M., Lemoine, R. & Ring, J. A. The use of DNA viruses as vectors for gene therapy. Gene Ther. 1, 367-384 (1994).
28. Gao, G.-P., Yang, Y. & Wilson, J. M. Biology of adenovirus vectors with E1 and E4 deletions for liver- directed gene therapy. J. Virol. 70, 8934-8943 (1996).
29. Dedieu, J.-F. et al. Long-term gene delivery into the livers of immunocompetent mice with E1/E4-defective adenoviruses. J. Virol. 71, 4626-4637 (1997).
30. Haecher, S. F. et al. In vivo expression of full-length human dystrophin from adenoviral vectors deleted of all viral genes. Hum. Gene Ther. 7, 1907-1914 (1996)
31. Lieber, A. et al. 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, 8944-8960 (1996).
32. Chen, H. H. et al. Persistence in muscle of an adenoviral vector that lacks all viral genes. Proc. Natl Acad. Sci. USA 94, 1645-1650 (1997).
33. Kaplan, J. M. et al. Characterization of factors involved in modulating persistence of transgene expression from recombinant adenovirus in the mouse lung. Hum. Gene Ther. 8, 45-56 (1997).
34. Ginsberg, H. S. et al. Role of early region 3 (E3) in pathogenesis of adenovirus disease. Proc. Natl Acad. Sci. USA 86, 3823-3827 (1989).
35. Elkon, K. B. et al. Tumor necrosis factor α plays a central role in immune-mediated clearance of adenoviral vectors. Proc. Natl Acad. Sci. USA 94, 9814-9819 (1997).
36. Kearns, W. G. et al. Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in a site- specific fashion in an immortalized epithelial cell line. Gene Ther. 3, 748-755 (1996).
37. Linden, R. M. & Berns, K. I. Site-specific integration by adeno-associated virus: a basis for a potential gene therapy vector. Gene Ther. 4, 4-5 (1997).
38. Miller, A. D. Putting muscle to work for gene therapy. Nature Med. 3, 278-279 (1997).
39. Xiao, X., Li, J. & Samulski, R. J. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J. Virol. 70, 8098-8108 (1996).
40. Fisher, K. J. et al. Recombinant adeno-associated virus for muscle directed gene therapy. Nature Med. 3, 306-312 (1997).
41. Breakefield, X. O. & DeLuca, N. A. Herpes simplex virus for gene delivery to neurons. New Biol. 3, 203-218 (1991).
42. Fink, D. J. & Glorioso, J. C. Engineering herpes simplex virus vectors for gene transfer to neurons. Nature Med. 3, 357-359 (1997).
43. Jacoby, D. R., Fraefel, C. & Breakefield, X. O. Hybrid vectors: a new generation of virus-based vectors designed to control the cellular fate of delivered genes. Gene Ther. 4, 1281-1283 (1997).
44. Bilbao, G. et al. Adenoviral/relroviral vector chimeras: a novel strategy to achieve high-efficiency stable transduction in vivo. FASEB J. 11, 624-634 (1997).
45. ORDA Report: Human Gene Therapy Protocols (2/3/98) (Office of Recombinant DNA Activities, NIH, Bethesda, MD, 1998).
46. Ram, Z. et al. Therapy of malignant brain tumors by intratumoral implantation of retroviral vector-producing cells. Nature Med 3, 1354-1361 (1997).
47. Oldfield, E. H. et al. Gene therapy for the treatment of brain tumors using intra-tumoral transduction with the thymidine kinase gene and intravenous ganciclovir. Hum. Gene Ther. 4, 39-69 (1993).
48. 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, 341-349 (1997).
49. esrv/rev retroviral vector) in HIV-1-infected subjects. Hum. Gene Ther. 6, 941-955 (1995).
50. Nabel, G. J. et al. Immune response in human melanoma after transfer of an allogeneic class I major histocompatibility complex gene with DNA-liposome complexes. Proc. Natl Acad. Sci. USA 93, 15388-15393 (1996).
51. Anderson, W. F. Human gene therapy. Science 256, 808-813 (1992).
52. Hoffman, R. M. et al. The feasibility of targeted selective gene therapy of the hair follicle. Nature Med. 1, 705-706 (1995).
53. Anderson, W. F. Human gene therapy: why draw a line? J. Med. Philos. 14, 681-693 (1989).
54. Fletcher, J. C. & Richter, G. Human fetal gene therapy: moral and ethical questions. Hum. Gene Ther. 7, 1605-1614 (1996).
55. Fletcher, J. C. & Anderson, W. F. Germ-line gene therapy: a new stage of debate. Law Med. Health Care 20, 26-39 (1992).
56. Schnieke, A. E. et al. Human factor IX transgenic sheep produced by transfer of nuclei from transfected fetal fibroblasts. Science 278, 2130-2133 (1997).