Adeno-associated virus as a vector for liver-directed gene therapy.
J Virol
; 72(12): 10222-6, 1998 Dec.
Article
en En
| MEDLINE
| ID: mdl-9811765
ABSTRACT
Factors relevant to the successful application of adeno-associated virus (AAV) vectors for liver-directed gene therapy were evaluated. Vectors with different promoters driving expression of human alpha-1-antitrypsin (alpha-1AT) were injected into the portal circulation of immunodeficient mice. alpha-1AT expression was stable but dependent on the promoter. Southern analysis of liver DNA revealed approximately 0.1 to 2.0 provirus copies/diploid genome in presumed head-to-tail concatamers. In situ hybridization and immunohistochemical analysis revealed expression in approximately 5% of hepatocytes clustered in the pericentral region. These results support the use of AAV as a vector for diseases treatable by targeting of hepatocytes.
Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Terapia Genética
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Dependovirus
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Vectores Genéticos
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Hígado
Tipo de estudio:
Risk_factors_studies
Límite:
Animals
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Humans
Idioma:
En
Año:
1998
Tipo del documento:
Article