| Autor: |
Montiel-Equihua CA; Centre for immunodeficiency, Molecular immunology Unit, UCL institute of Child Health, London, UK., Thrasher AJ, Gaspar HB |
| Jazyk: |
angličtina |
| Zdroj: |
Stem cells and cloning : advances and applications [Stem Cells Cloning] 2009 Dec 22; Vol. 3, pp. 1-12. Date of Electronic Publication: 2009 Dec 22. |
| DOI: |
10.2147/sccaa.s5570 |
| Abstrakt: |
The history of stem cell gene therapy is strongly linked to the development of gene therapy for severe combined immunodeficiencies (SCID) and especially adenosine deaminase (ADA)-deficient SCID. Here we discuss the developments achieved in over two decades of clinical and laboratory research that led to the establishment of a protocol for the autologous transplant of retroviral vector-mediated gene-modified hematopoietic stem cells, which has proved to be both successful and, to date, safe. Patients in trials in three different countries have shown long-term immunological and metabolic correction. Nevertheless, improvements to the safety profile of viral vectors are underway and will undoubtedly reinforce the position of stem cell gene therapy as a treatment option for ADA-SCID. |
| Databáze: |
MEDLINE |
| Externí odkaz: |
|
