Effective treatment of familial hypercholesterolaemia in the mouse model using adenovirus-mediated transfer of the VLDL receptor gene.
Nat Genet
; 13(1): 54-62, 1996 May.
Article
en En
| MEDLINE
| ID: mdl-8673104
ABSTRACT
Liver directed gene transfer with adenoviral vectors is being considered for the treatment of several metabolic diseases, including familial hypercholesterolaemia (FH). Gene replacement therapy of human low density lipoprotein (LDL) receptor gene into the murine model of FH transiently corrected the dyslipidaemia; however, humoral and cellular immune responses to LDL receptor developed--possibly contributing to the associated hepatitis and extinguishing of transgene expression. We evaluated an alternative strategy of ectopic expression in the liver of the very low density lipoprotein (VLDL) receptor, which is homologous to the LDL receptor but has a different pattern of expression. Infusion of recombinant adenoviruses containing the VLDL receptor gene corrected the dsylipidaemia in the FH mouse and circumvented immune responses to the transgene leading to a more prolonged metabolic correction.
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Base de datos:
MEDLINE
Asunto principal:
Receptores de LDL
/
Terapia Genética
/
Técnicas de Transferencia de Gen
/
Citomegalovirus
/
Hiperlipoproteinemia Tipo II
/
Hígado
Límite:
Animals
/
Humans
Idioma:
En
Revista:
Nat Genet
Asunto de la revista:
GENETICA MEDICA
Año:
1996
Tipo del documento:
Article
País de afiliación:
Estados Unidos