Gaucher's disease: studies of gene transfer to haematopoietic cells.
Baillieres Clin Haematol
; 10(4): 765-78, 1997 Dec.
Article
en En
| MEDLINE
| ID: mdl-9497863
ABSTRACT
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.
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Bases de datos:
MEDLINE
Asunto principal:
Terapia Genética
/
Enfermedad de Gaucher
Límite:
Humans
Idioma:
En
Revista:
Baillieres Clin Haematol
Asunto de la revista:
HEMATOLOGIA
Año:
1997
Tipo del documento:
Article
País de afiliación:
Estados Unidos