Abstract
Advances in the understanding of molecular biology of human disease and the development of efficient gene transfer techniques have resulted in practical approaches to human gene therapy, with new techniques being developed at an increasing rate. The first trials have now begun in humans and initial results are positive.
This is a preview of subscription content, log in via an institution to check access.
Similar content being viewed by others
References
Hum. Gene Ther. 1, 93–103 (1990).
Hum. Gene Ther. 2, 251–256 (1991).
Rosenberg, S. A. Cancer Res. 51 (suppl.), 5074–5079 (1991).
Rosenberg, S. A. et al. New Engl. J. Med. 323, 570–578 (1990).
Lupton, S. D., Brunton, L. L., Kalberg, V. A. & Overell, R. W. Molec. cell. Biol. 11, 3374–3378 (1991).
Chowdhury, J. R. et al. Science 254, 1802–1805 (1991).
Tepper, R. I., Pattengale, P. K. & Leder, P. Cell 57, 503–512 (1989).
Watanabe, Y. et al. Proc. natn. Acad. Sci. U.S.A. 85, 9456–9460 (1989).
Fearon, E. R. et al. Cell 60, 397–403 (1990).
Gansbacher, B. et al. J. exp. Med. 172, 1217–1224 (1990).
Gansbacher, B. et al. Cancer Res. 50, 7820–7825 (1990).
Colombo, M. P. et al. J. exp. Med. 173, 889–897 (1991).
Asher, A. L. et al. J. Immun. 146, 3227–3234 (1991).
Golumbek, P. T. et al. Science 254, 713–716 (1991).
Miller, D. G., Adam, M. A. & Miller, A. D. Molec. cell. Biol. 10, 4239–4242 (1990).
Scaden, D. T., Fuller, B. & Cunningham, J. M. J. Virol. 64, 424–427 (1990).
Kotin, R. M. et al. Proc. natn. Acad. Sci. U.S.A. 87, 2211–2215 (1990).
Samulski, R. J. et al. EMBO J. 10, 3941–3950 (1991).
Miller, A. D. Hum. Gene Ther. 1, 5–14 (1990).
Miller, A. D. & Buttimore, C. Molec. cell. Biol. 6, 2895–2902 (1986).
Miller, A. D. & Rosman, G. J. Biotechniques 7, 980–990 (1989).
Lynch, C. M. & Miller, A. D. J. Virol. 65, 3887–3890 (1991).
Kolberg, R. J. NIH Res. 4, 43–44 (1992).
Cornetta, K. et al. Hum. Gene Ther. 2, 215–219 (1991).
Rosenfeld, M. A. et al. Cell 68, 143–155 (1992).
Wu, G. Y. et al. J. biol. Chem. 266, 14338–14342 (1991).
Zenke, M. et al. Proc. natn. Acad. Sci. U.S.A. 87, 3655–3659 (1990).
Curiel, D. T., Agarwal, S., Wagner, E. & Cotten, M. Proc. natn. Acad. Sci. U.S.A. 88, 8850–8854 (1991).
Kantoff, P. W. et al. J. exp. Med. 166, 219–234 (1987).
Bodine, D. M. et al. Proc. natn. Acad. Sci. U.S.A. 87, 3738–3742 (1990).
Stead, R. B., Kwok, W. W., Storb, R. & Miller, A. D. Blood 71, 742–747 (1988).
Schuening, F. G. et al. Blood 78, 2568–2576 (1991).
Lothrop, C. D. et al. Blood 78, 237–245 (1991).
Kantoff, P. W. et al. Blood 73, 1066–1073 (1989).
Wolff, J. A. et al. Science 247, 1465–1468 (1990).
Wolff, J. A. et al. Biotechniques 11, 474–485 (1991).
Jiao, S. et al. Hum. Gene Ther. 3, 21–33 (1992).
Barr, E. & Leiden, J. M. Science 254, 1507–1509 (1991).
Dhawan, J. et al. Science 254, 1509–1512 (1991).
Palmer, T. D., Thompson, A. R. & Miller, A. D. Blood 73, 438–445 (1989).
Palmer, T. D., Rosman, G. J., Osborne, W. R. A. & Miller, A. D. Proc. natn. Acad. Sci. U.S.A. 88, 1330–1334 (1991).
Lynch, C. M. et al. Proc. natn. Acad. Sci. U.S.A. 89, 1138–1142 (1992).
Sullenger, B. A., Gallardo, H. F., Ungers, G. E. & Gilboa, E. Cell 63, 601–608 (1990).
Hum. Gene Ther. 2, 331–358 (1991).
Cornetta, K. et al. Hum. Gene Ther. 1, 15–30 (1990).
Miller, A. D. Blood 76, 271–278 (1990).
Fraser, C. C., Szilvassy, S. J., Eaves, C. J. & Humphries, R. K. Proc. natn. Acad. Sci. U.S.A. 89, 1968–1972 (1992).
Author information
Authors and Affiliations
Rights and permissions
About this article
Cite this article
Miller, A. Human gene therapy comes of age. Nature 357, 455–460 (1992). https://doi.org/10.1038/357455a0
Issue Date:
DOI: https://doi.org/10.1038/357455a0
- Springer Nature Limited
This article is cited by
-
Application of Genetic Engineering in Biotherapeutics Development
Journal of Pharmaceutical Innovation (2020)
-
Gene therapy for hemophilia B mice with scAAV8-LP1-hFIX
Frontiers of Medicine (2016)
-
A Broad Overview and Review of CRISPR-Cas Technology and Stem Cells
Current Stem Cell Reports (2016)