Efficient retrovirus-mediated transfer of the multidrug resistance 1 gene into autologous human long-term repopulating hematopoietic stem cells.
Nat Med
; 6(6): 652-8, 2000 Jun.
Article
em En
| MEDLINE
| ID: mdl-10835681
ABSTRACT
Pre-clinical studies indicate that efficient retrovirus-mediated gene transfer into hematopoietic stem cells and progenitor cells can be achieved by co-localizing retroviral particles and target cells on specific adhesion domains of fibronectin. In this pilot study, we used this technique to transfer the human multidrug resistance 1 gene into stem and progenitor cells of patients with germ cell tumors undergoing autologous transplantation. There was efficient gene transfer into stem and progenitor cells in the presence of recombinant fibronectin fragment CH-296. The infusion of these cells was associated with no harmful effects and led to prompt hematopoietic recovery. There was in vivo vector expression, but it may have been limited by the high rate of aberrant splicing of the multidrug resistance 1 gene in the vector. Gene marking has persisted more than a year at levels higher than previously reported in humans.
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Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Retroviridae
/
Células-Tronco Hematopoéticas
/
Fibronectinas
/
Germinoma
/
Técnicas de Transferência de Genes
/
Transplante de Células-Tronco Hematopoéticas
/
Genes MDR
/
Vetores Genéticos
Tipo de estudo:
Observational_studies
/
Prognostic_studies
/
Risk_factors_studies
Limite:
Adolescent
/
Adult
/
Humans
/
Middle aged
Idioma:
En
Revista:
Nat Med
Assunto da revista:
BIOLOGIA MOLECULAR
/
MEDICINA
Ano de publicação:
2000
Tipo de documento:
Article
País de afiliação:
Estados Unidos