Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA − Immunodeficient Patients
10.1126/science.270.5235.470
Crossref journal-article
American Association for the Advancement of Science (AAAS)
Science (221)
Abstract

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.

Bibliography

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, 270(5235), 470–475.

Authors 12
  1. Claudio Bordignon (first)
  2. Luigi D. Notarangelo (additional)
  3. Nadia Nobili (additional)
  4. Giuliana Ferrari (additional)
  5. Giulia Casorati (additional)
  6. Paola Panina (additional)
  7. Evelina Mazzolari (additional)
  8. Daniela Maggioni (additional)
  9. Claudia Rossi (additional)
  10. Paolo Servida (additional)
  11. Alberto G. Ugazio (additional)
  12. Fulvio Mavilio (additional)
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Dates
Type When
Created 18 years, 10 months ago (Oct. 27, 2006, 2:19 p.m.)
Deposited 1 year, 7 months ago (Jan. 12, 2024, 4:19 p.m.)
Indexed 1 week, 5 days ago (Aug. 23, 2025, 1:02 a.m.)
Issued 29 years, 10 months ago (Oct. 20, 1995)
Published 29 years, 10 months ago (Oct. 20, 1995)
Published Print 29 years, 10 months ago (Oct. 20, 1995)
Funders 0

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@article{Bordignon_1995, title={Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA − Immunodeficient Patients}, volume={270}, ISSN={1095-9203}, url={http://dx.doi.org/10.1126/science.270.5235.470}, DOI={10.1126/science.270.5235.470}, number={5235}, journal={Science}, publisher={American Association for the Advancement of Science (AAAS)}, author={Bordignon, Claudio and Notarangelo, Luigi D. and Nobili, Nadia and Ferrari, Giuliana and Casorati, Giulia and Panina, Paola and Mazzolari, Evelina and Maggioni, Daniela and Rossi, Claudia and Servida, Paolo and Ugazio, Alberto G. and Mavilio, Fulvio}, year={1995}, month=oct, pages={470–475} }