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T Lymphocyte-Directed Gene Therapy for ADA − SCID: Initial Trial Results After 4 Years
Abstract
In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase (ADA) gene into the T cells of two children with severe combined immunodeficiency (ADA − SCID). The number of blood T cells normalized as did many cellular and humoral immune responses. Gene treatment ended after 2 years, but integrated vector and ADA gene expression in T cells persisted. Although many components remain to be perfected, it is concluded here that gene therapy can be a safe and effective addition to treatment for some patients with this severe immunodeficiency disease.
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Citations
Cited by
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Pack, D. W., Hoffman, A. S., Pun, S., & Stayton, P. S. (2005) · Nature Reviews Drug Discovery · doi:10.1038/nrd1775 · Cited by 2,568
Naldini, L., Blömer, U., Gage, F. H., Trono, D., & Verma, I. M. (1996) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.93.21.11382 · Cited by 1,499
The current landscape of nucleic acid therapeutics
Kulkarni, J. A., Witzigmann, D., Thomson, S. B., Chen, S., Leavitt, B. R., Cullis, P. R., & van der Meel, R. (2021) · Nature Nanotechnology · doi:10.1038/s41565-021-00898-0 · Cited by 1,371
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
Aiuti, A., Slavin, S., Aker, M., Ficara, F., Deola, S., Mortellaro, A., Morecki, S., Andolfi, G., Tabucchi, A., Carlucci, F., et al. (2002) · Science · doi:10.1126/science.1070104 · Cited by 975
GENE THERAPY: Twenty-First Century Medicine
Verma, I. M., & Weitzman, M. D. (2005) · Annual Review of Biochemistry · doi:10.1146/annurev.biochem.74.050304.091637 · Cited by 616
Metadata
- Authors
- Blaese, R. M., Culver, K. W., Miller, A. D., Carter, C. S., Fleisher, T., Clerici, M., Shearer, G., Chang, L., Chiang, Y., Tolstoshev, P., Greenblatt, J. J., Rosenberg, S. A., Klein, H., Berger, M., Mullen, C. A., Ramsey, W. J., Muul, L., Morgan, R. A., & Anderson, W. F.
- Venue
- Science
- Published
- 1995-10-20
- Volume
- 270
- Issue
- 5235
- Pages
- 475-480
- ISSN
- 0036-8075 (ISSN-L)
- 1095-9203
- OpenAlex
- W2032678451
- Cited by
- 1,550
- References
- 0
- FWCI
- 36.91
- Type
- journal article
- Language
- en
- Open access
- closed
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- CoChat. (2026). Citation check: T Lymphocyte-Directed Gene Therapy for ADA − SCID: Initial Trial Results After 4 Years. https://app.cochat.ai/papers/10.1126/science.270.5235.475
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- Blaese, R. M., Culver, K. W., Miller, A. D., Carter, C. S., Fleisher, T., Clerici, M., Shearer, G., Chang, L., Chiang, Y., Tolstoshev, P., Greenblatt, J. J., Rosenberg, S. A., Klein, H., Berger, M., Mullen, C. A., Ramsey, W. J., Muul, L., Morgan, R. A., & Anderson, W. F. (1995). T Lymphocyte-Directed Gene Therapy for ADA − SCID: Initial Trial Results After 4 Years. Science. https://doi.org/10.1126/science.270.5235.475
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