Gaucher's disease: studies of gene transfer to haematopoietic cells.
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.
Barranger
J A
JA
University of Pittsburgh Medical Center, PA 15261, USA.
Rice
E O
EO
Dunigan
J
J
Sansieri
C
C
Takiyama
N
N
Beeler
M
M
Lancia
J
J
Lucot
S
S
Scheirer-Fochler
S
S
Mohney
T
T
Swaney
W
W
Bahnson
A
A
Ball
E
E
D005776:Gaucher Disease / Q000235:genetics / Q000628:therapy*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D006412:Hematopoietic Stem Cells / Q000502:physiology; D006801:Humans