| Autor: |
J A, Barranger, E O, Rice, J, Dunigan, C, Sansieri, N, Takiyama, M, Beeler, J, Lancia, S, Lucot, S, Scheirer-Fochler, T, Mohney, W, Swaney, A, Bahnson, E, Ball |
| Rok vydání: |
1998 |
| Předmět: |
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| Zdroj: |
Bailliere's clinical haematology. 10(4) |
| ISSN: |
0950-3536 |
| Popis: |
Transfer of the gene coding for glucocerebrosidase (GC) via a retroviral vector (MFG-GC) to haematopoietic progenitors results in engraftment and life-long expression of the human protein at high levels in transplanted mice. Studies of human CD34 cells were carried out to evaluate their potential use in a gene therapy approach to Gaucher's disease. High transduction efficiency and correction of the enzyme deficiency was possible in CD34 cells obtained from patients with Gaucher's disease. Based on these results, a clinical trial of gene therapy was designed and initiated. Preliminary results of this study indicate the persistence or engraftment of genetically corrected cells in the transplanted patients. |
| Databáze: |
OpenAIRE |
| Externí odkaz: |
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