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