Note to users. If you're seeing this message, it means that your browser cannot find this page's style/presentation instructions -- or possibly that you are using a browser that does not support current Web standards. Find out more about why this message is appearing, and what you can do to make your experience of our site the best it can be.
SNM Organization

Site Tools

  • AAAS
  • Subscribe
  • Feedback

Site Search

Search Advanced

Science 26 October 1984:
Vol. 226. no. 4673, pp. 401 - 409
DOI: 10.1126/science.6093246

Articles

Science, Vol 226, Issue 4673, 401-409
Copyright © 1984 by American Association for the Advancement of Science


articles

Prospects for human gene therapy

WF Anderson

Procedures have now been developed for inserting functional genes into the bone marrow of mice. The most effective delivery system at present uses retroviral-based vectors to transfer a gene into murine bone marrow cells in culture. The genetically altered bone marrow is then implanted into recipient animals. These somatic cell gene therapy techniques are becoming increasingly efficient. Their future application in humans should result in at least partial correction of a number of genetic disorders. However, the safety of the procedures must still be established by further animal studies before human clinical trials would be ethical.


THIS ARTICLE HAS BEEN CITED BY OTHER ARTICLES:
Development of gene therapy for blood disorders.
A. W. Nienhuis (2008)
Blood 111, 4431-4444
   Abstract »    Full Text »    PDF »
Gene therapy in clinical medicine.
S M Selkirk (2004)
Postgrad. Med. J. 80, 560-570
   Abstract »    Full Text »    PDF »
Regulated Gene Expression in Gene Therapy.
P. W. ZOLTICK and J. M. WILSON (2001)
Ann. N.Y. Acad. Sci. 953, 53-63
   Abstract »    Full Text »    PDF »
Development of a Stable Retrovirus Vector Capable of Long-Term Expression of {gamma}-Globin mRNA in Mouse Erythrocytes.
D. E. SABATINO, N. E. SEIDEL, A. P. CLINE, S. M. ANDERSON, P. G. GALLAGHER, and D. M. BODINE (2001)
Ann. N.Y. Acad. Sci. 938, 246-261
   Abstract »    Full Text »    PDF »
RD114-Pseudotyped Oncoretroviral Vectors: Biological and Physical Properties.
P. F. KELLY, J. CARRINGTON, A. NATHWANI, and E. F. VANIN (2001)
Ann. N.Y. Acad. Sci. 938, 262-277
   Abstract »    Full Text »    PDF »
Lentivirus-based vectors transduce mouse hematopoietic stem cells with similar efficiency to Moloney murine leukemia virus-based vectors.
S. Barrette, J. L. Douglas, N. E. Seidel, and D. M. Bodine (2000)
Blood 96, 3385-3391
   Abstract »    Full Text »    PDF »
Highly efficient gene transfer into cord blood nonobese diabetic/severe combined immunodeficiency repopulating cells by oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein.
P. F. Kelly, J. Vandergriff, A. Nathwani, A. W. Nienhuis, and E. F. Vanin (2000)
Blood 96, 1206-1214
   Abstract »    Full Text »    PDF »
HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells.
N. Uchida, R. E. Sutton, A. M. Friera, D. He, M. J. Reitsma, W. C. Chang, G. Veres, R. Scollay, and I. L. Weissman (1998)
PNAS 95, 11939-11944
   Abstract »    Full Text »    PDF »
Improved Amphotropic Retrovirus-Mediated Gene Transfer into Hematopoietic Stem Cells.
D. M. BODINE, C. E. DUNBAR, L. J. GIRARD, N. E. SEIDEL, A. P. CLINE, R. E. DONAHUE, and D. ORLIC (1998)
Ann. N.Y. Acad. Sci. 850, 139-150
   Abstract »    Full Text »    PDF »
Identification of Human and Mouse Hematopoietic Stem Cell Populations Expressing High Levels of mRNA Encoding Retrovirus Receptors.
D. Orlic, L. J. Girard, S. M. Anderson, L. C. Pyle, M. C. Yoder, H. E. Broxmeyer, and D. M. Bodine (1998)
Blood 91, 3247-3254
   Abstract »    Full Text »    PDF »
Human gene therapy.
W. Anderson (1992)
Science 256, 808-813
   Abstract »    PDF »
An in vivo model of somatic cell gene therapy for human severe combined immunodeficiency.
G Ferrari, S Rossini, R Giavazzi, D Maggioni, N Nobili, M Soldati, G Ungers, F Mavilio, E Gilboa, and C Bordignon (1991)
Science 251, 1363-1366
   Abstract »    PDF »
Retrovirus vectors: promise and reality.
H. Temin (1989)
Science 246, 983
   PDF »
Evidence for a complex regulatory array in the first intron of the human adenosine deaminase gene..
B Aronow, D Lattier, R Silbiger, M Dusing, J Hutton, G Jones, J Stock, J McNeish, S Potter, and D Witte (1989)
Genes & Dev. 3, 1384-1400
   Abstract »    PDF »
Progress toward human gene therapy.
T Friedmann (1989)
Science 244, 1275-1281
   Abstract »    PDF »
High-level recombinant gene expression in rabbit endothelial cells transduced by retroviral vectors.
J. Zwiebel, S. Freeman, P. Kantoff, K Cornetta, U. Ryan, and W. Anderson (1989)
Science 243, 220-222
   Abstract »    PDF »
Clonal gene therapy: transplanted mouse fibroblast clones express human alpha 1-antitrypsin gene in vivo.
R. Garver Jr, A Chytil, M Courtney, and R. Crystal (1987)
Science 237, 762-764
   Abstract »    PDF »
Implantation of genetically engineered fibroblasts into mice: implications for gene therapy.
R. Selden, M. Skoskiewicz, K. Howie, P. Russell, and H. Goodman (1987)
Science 236, 714-718
   Abstract »    PDF »
Review Article: The Inherited Neurodegenerative Disorders of Childhood: Clinical Assessment.
A. K. Percy (1987)
J Child Neurol 2, 82-97
   Abstract »    PDF »
Correction of murine beta-thalassemia by gene transfer into the germ line.
F Costantini, K Chada, and J Magram (1986)
Science 233, 1192-1194
   Abstract »    PDF »
The application of bone marrow transplantation to the treatment of genetic diseases.
R Parkman (1986)
Science 232, 1373-1378
   Abstract »    PDF »
Gene therapy--so near and yet so far away.
J. Marx (1986)
Science 232, 824-825
   PDF »
Gene expression in mice after high efficiency retroviral-mediated gene transfer.
M. Eglitis, P Kantoff, E Gilboa, and W. Anderson (1985)
Science 230, 1395-1398
   Abstract »    PDF »
Retroviral vector-mediated gene transfer into human hematopoietic progenitor cells.
H. Gruber, K. Finley, R. Hershberg, S. Katzman, P. Laikind, J. Seegmiller, T Friedmann, J. Yee, and D. Jolly (1985)
Science 230, 1057-1061
   Abstract »    PDF »
Gene therapy: research in public.
B. Culliton (1985)
Science 227, 493-496
   PDF »
Long-term expression of gamma -globin mRNA in mouse erythrocytes from retrovirus vectors containing the human gamma -globin gene fused to the ankyrin-1 promoter.
D. E. Sabatino, N. E. Seidel, G. J. Aviles-Mendoza, A. P. Cline, S. M. Anderson, P. G. Gallagher, and D. M. Bodine (2000)
PNAS 97, 13294-13299
   Abstract »    Full Text »    PDF »



ADVERTISEMENT
Click Me!

ADVERTISEMENT

To Advertise     Find Products


Science. ISSN 0036-8075 (print), 1095-9203 (online)