Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency
University of Rome Tor Vergata · The San Raffaele Telethon Institute for Gene Therapy · +20 more institutions
Abstract
We investigated the long-term outcome of gene therapy for severe combined immunodeficiency (SCID) due to the lack of adenosine deaminase (ADA), a fatal disorder of purine metabolism and immunodeficiency.
We infused autologous CD34+ bone marrow cells transduced with a retroviral vector containing the ADA gene into 10 children with SCID due to ADA deficiency who lacked an HLA-identical sibling donor, after nonmyeloablative conditioning with busulfan. Enzyme-replacement therapy was not given after infusion of the cells.
Citation impact
- FWCI
- 71.47
- Percentile
- 100%
- References
- 35
Authors
30- AAAlessandro AiutiCorresponding
University of Rome Tor Vergata, The San Raffaele Telethon Institute for Gene Therapy
- FCFederica Cattaneo
University of Siena, San Raffaele University of Rome
- SGStefania Galimberti
University of Milano-Bicocca
- UBUlrike Benninghoff
Fondazione Telethon
- BCBarbara Cassani
Fondazione Humanitas per la Ricerca
Topics & keywords
- Adenosine deaminase deficiency
- Medicine
- Adenosine deaminase
- Genetic enhancement
- Severe combined immunodeficiency
- Bone marrow
- Immunology
- Enzyme replacement therapy
- Good health and well-being