In vivo site-directed mutagenesis of the factor IX gene by chimeric RNA/DNA oligonucleotides.
Nat Med
; 4(3): 285-90, 1998 Mar.
Article
en En
| MEDLINE
| ID: mdl-9500600
A chimeric RNA/DNA oligonucleotide was constructed to induce a sequence mutation in the rat factor IX gene, resulting in prolonged coagulation. Oligonucleotides were targeted to hepatocytes in cell culture or in vivo by intravenous injection. Nucleotide conversion was both site-specific and dose-dependent. The mutated gene was associated in vivo with significantly reduced factor IX coagulant activity and a marked prolongation of the activated partial thromboplastin time. The results demonstrate that single base-pair alterations can be introduced in hepatocytes in situ by RNA/DNA oligonucleotides, suggesting a potentially powerful strategy for hepatic gene repair without the use of viral vectors.
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Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Oligonucleótidos
/
Factor IX
/
Mutagénesis Sitio-Dirigida
/
Marcación de Gen
/
Hígado
Límite:
Animals
Idioma:
En
Revista:
Nat Med
Asunto de la revista:
BIOLOGIA MOLECULAR
/
MEDICINA
Año:
1998
Tipo del documento:
Article
País de afiliación:
Estados Unidos