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Titolo:
Introduction of single base substitutions at homologous chromosomal sequences by adeno-associated virus vectors
Autore:
Inoue, N; Dong, R; Hirata, RK; Russell, DW;
Indirizzi:
Univ Washington, Div Hematol, Seattle, WA 98195 USA Univ Washington Seattle WA USA 98195 , Div Hematol, Seattle, WA 98195 USA
Titolo Testata:
MOLECULAR THERAPY
fascicolo: 4, volume: 3, anno: 2001,
pagine: 526 - 530
SICI:
1525-0016(200104)3:4<526:IOSBSA>2.0.ZU;2-7
Fonte:
ISI
Lingua:
ENG
Soggetto:
HYPOXANTHINE PHOSPHORIBOSYLTRANSFERASE GENE; APE LEUKEMIA-VIRUS; NUCLEAR EXTRACTS; HUMAN-CELLS; MUTATIONS; MISPAIRS;
Keywords:
AAV; mutation; gene targeting; mismatch; substitution; vector;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
22
Recensione:
Indirizzi per estratti:
Indirizzo: Russell, DW Univ Washington, Div Hematol, Mail Stop 357720,Room K-236A,HSB, Seattle, WA 98195 USA Univ Washington Mail Stop 357720,Room K-236A,HSB Seattle WA USA 98195
Citazione:
N. Inoue et al., "Introduction of single base substitutions at homologous chromosomal sequences by adeno-associated virus vectors", MOL THER, 3(4), 2001, pp. 526-530

Abstract

Adeno-associated virus (AAV) vectors can modify homologous chromosomal sequences at high rates. This gene targeting transduction pathway is distinct from the integrating and episomal pathways used in gene addition approaches. In previous studies, AAV vectors were used to introduce small insertion and deletion mutations at homologous chromosomal loci. Here we show that AAV-mediated gene targeting can also be used to introduce all possible types of single base substitution mutations at the endogenous single-copy hypoxanthine phosphoribosyl transferase locus. Southern blot and sequence analysis showed that the point mutations were introduced with high fidelity. We alsoshow that AAV vectors can repair chromosomal alkaline phosphatase genes containing point mutations. Our results suggest that AAV vectors can be used to introduce single base substitutions at high frequencies in normal human cells, including the correction of point mutations responsible for genetic diseases.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 04/07/20 alle ore 18:07:25