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Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA − Immunodeficient Patients
Open accessAbstract
Adenosine deaminase (ADA) deficiency results in severe combined immunodeficiency, the first genetic disorder treated by gene therapy. Two different retroviral vectors were used to transfer ex vivo the human ADA minigene into bone marrow cells and peripheral blood lymphocytes from two patients undergoing exogenous enzyme replacement therapy. After 2 years of treatment, long-term survival of T and B lymphocytes, marrow cells, and granulocytes expressing the transferred ADA gene was demonstrated and resulted in normalization of the immune repertoire and restoration of cellular and humoral immunity. After discontinuation of treatment, T lymphocytes, derived from transduced peripheral blood lymphocytes, were progressively replaced by marrow-derived T cells in both patients. These results indicate successful gene transfer into long-lasting progenitor cells, producing a functional multilineage progeny.
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Citations
Cited by
Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease
Cavazzana-Calvo, M., Hacein-Bey, S., Basile, G. D. S., Gross, F., Yvon, E., Nusbaum, P., Selz, F., Hue, C., Certain, S., Casanova, J.-L., et al. (2000) · Science · doi:10.1126/science.288.5466.669 · Cited by 2,167
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
Gene therapy clinical trials worldwide to 2017: An update
Ginn, S. L., Amaya, A. K., Alexander, I. E., Edelstein, M., & Abedi, M. R. (2018) · The Journal of Gene Medicine · doi:10.1002/jgm.3015 · Cited by 903
Wirth, T., Parker, N., & Ylä-Herttuala, S. (2013) · Gene · doi:10.1016/j.gene.2013.03.137 · Cited by 607
High, K. A., & Roncarolo, M. G. (2019) · New England Journal of Medicine · doi:10.1056/nejmra1706910 · Cited by 555
Metadata
- Authors
- Bordignon, C., Notarangelo, L. D., Nobili, N., Ferrari, G., Casorati, G., Panina, P., Mazzolari, E., Maggioni, D., Rossi, C., Servida, P., Ugazio, A. G., & Mavilio, F.
- Venue
- Science
- Published
- 1995-10-20
- Volume
- 270
- Issue
- 5235
- Pages
- 470-475
- ISSN
- 0036-8075 (ISSN-L)
- 1095-9203
- OpenAlex
- W2066271426
- Cited by
- 832
- References
- 0
- FWCI
- 31.64
- Type
- journal article
- Language
- en
- Open access
- green
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- CoChat. (2026). Citation check: Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA − Immunodeficient Patients. https://app.cochat.ai/papers/10.1126/science.270.5235.470
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- Bordignon, C., Notarangelo, L. D., Nobili, N., Ferrari, G., Casorati, G., Panina, P., Mazzolari, E., Maggioni, D., Rossi, C., Servida, P., Ugazio, A. G., & Mavilio, F. (1995). Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA − Immunodeficient Patients. Science. https://doi.org/10.1126/science.270.5235.470
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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