The DOI matches its Crossref record.
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
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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
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Somia, N., & Verma, I. M. (2000) · Nature Reviews Genetics · doi:10.1038/35038533 · Cited by 554
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Self-citationLiu, J. M., Kim, S., Read, E. J., Futaki, M., Dokal, I., Carter, C. S., Leitman, S. F., Pensiero, M., Young, N. S., & Walsh, C. E. (1999) · Human Gene Therapy · doi:10.1089/10430349950016988 · Cited by 117
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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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