In vivo selection using a cell-growth switch.
Nat Genet
; 26(1): 64-6, 2000 Sep.
Article
en En
| MEDLINE
| ID: mdl-10973250
ABSTRACT
A major obstacle to stem-cell gene therapy rests in the inability to deliver a gene into a therapeutically relevant fraction of stem cells. One way to circumvent this obstacle is to use selection. Vectors containing two linked genes serve as the basis for selection, with one gene encoding a selectable product and the other, a therapeutic protein. Applying selection in vivo has the potential to bring a minor population of genetically corrected cells into the therapeutic range. But strategies for achieving in vivo selection have traditionally relied on genes that confer resistance to cytotoxic drugs and are encumbered by toxicity. Here we describe a new system for in vivo selection that uses a 'cell-growth switch', allowing a minor population of genetically corrected cells into the therapeutic range. But strategies for achieving in vivo selection have traditionally relied on genes that confer resistance to cytotoxic drugs and are encumbered by toxicity. Here we describe a new system for in vivo selection that uses a 'cell-growth switch', allowing a minor population of genetically modified cells to be inducibly amplified, thereby averting the risks associated with cytotoxic drugs. This system provides a general platform for conditionally expanding genetically modified cell populations in vivo, and may have widespread applications in gene and cell therapy.
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Banco de datos:
MEDLINE
Asunto principal:
Terapia Genética
/
Separación Celular
/
Receptores de Citocinas
/
Vectores Genéticos
/
Proteínas de Neoplasias
Idioma:
En
Año:
2000
Tipo del documento:
Article