Abstract
In the last decade, gene therapy for adenosine deaminase deficiency has been developed as a successful alternative strategy to allogeneic bone marrow transplant and enzyme replacement therapy. Infusion of autologous hematopoietic stem cells, corrected ex vivo by retroviral vectors and combined to low-intensity conditioning regimen, has resulted in immunologic improvement, metabolic correction, and long-term clinical benefits. These findings have opened the way to applications of gene therapy in other primary immune deficiencies using novel vector technology.
Original language | English |
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Pages (from-to) | 249-260 |
Number of pages | 12 |
Journal | Immunology and Allergy Clinics of North America |
Volume | 30 |
Issue number | 2 |
DOIs | |
Publication status | Published - 2010 |
Keywords
- Adenosine deaminase
- Gene therapy
- Haematopoietic stem cell
- Immune deficiency
- Reduced-intensity conditioning regimen
ASJC Scopus subject areas
- Immunology and Allergy
- Immunology
- Medicine(all)