Long-term cure of the photosensitivity of murine erythropoietic protoporphyria by preselective gene therapy.
Nat Med
; 5(7): 768-73, 1999 Jul.
Article
en En
| MEDLINE
| ID: mdl-10395321
Definitive cure of an animal model of a human disease by gene transfer into hematopoietic stem cells has not yet been accomplished in the absence of spontaneous in vivo selection for transduced cells. Erythropoietic protoporphyria is a genetic disease in which ferrochelatase is defective. Protoporphyrin accumulates in erythrocytes, leaks into the plasma and results in severe skin photosensitivity. Using a mouse model of erythropoietic protoporphyria, we demonstrate here that ex vivo preselection of hematopoietic stem cells transduced with a polycistronic retrovirus expressing both human ferrochelatase and green fluorescent protein results in complete and long-term correction of skin photosensitivity in all transplanted mice.
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Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Trastornos por Fotosensibilidad
/
Terapia Genética
/
Trasplante de Médula Ósea
/
Porfiria Hepatoeritropoyética
/
Ferroquelatasa
Límite:
Animals
/
Humans
/
Male
Idioma:
En
Revista:
Nat Med
Asunto de la revista:
BIOLOGIA MOLECULAR
/
MEDICINA
Año:
1999
Tipo del documento:
Article
País de afiliación:
Estados Unidos
Pais de publicación:
Estados Unidos