Precise hit: Adeno-associated virus in gene targeting

被引:86
作者
Vasileva, A
Jessberger, R [1 ]
机构
[1] Univ Technol Dresden, Inst Psyshiol Chem, Dresden, Germany
[2] Mt Sinai Sch Med, Dept Gene & Cell Med, New York, NY USA
关键词
D O I
10.1038/nrmicro1266
中图分类号
Q93 [微生物学];
学科分类号
071005 ; 100705 ;
摘要
Vectors based on the adeno-associated virus (AAV) have attracted much attention as potent gene-delivery vehicles, mainly because of the persistence of this non-pathogenic virus in the host cell and its sustainable therapeutic gene expression. However, virus infection can be accompanied by potentially mutagenic random vector integration into the genome. A novel approach to AAV-mediated gene therapy based on gene targeting through homologous recombination allows efficient, high-fidelity, non-mutagenic gene repair in a host cell.
引用
收藏
页码:837 / 847
页数:11
相关论文
共 87 条
[1]   DNA-DAMAGING AGENTS GREATLY INCREASE THE TRANSDUCTION OF NONDIVIDING CELLS BY ADENOASSOCIATED VIRUS VECTORS [J].
ALEXANDER, IE ;
RUSSELL, DW ;
MILLER, AD .
JOURNAL OF VIROLOGY, 1994, 68 (12) :8282-8287
[2]   Adult mesenchymal stem cells: characterization, differentiation, and application in cell and gene therapy [J].
Baksh, D ;
Song, L ;
Tuan, RS .
JOURNAL OF CELLULAR AND MOLECULAR MEDICINE, 2004, 8 (03) :301-316
[3]   Side effects of retroviral gene transfer into hematopoietic stem cells [J].
Baum, C ;
Düllmann, J ;
Li, ZX ;
Fehse, B ;
Meyer, J ;
Williams, DA ;
von Kalle, C .
BLOOD, 2003, 101 (06) :2099-2114
[4]   Recombinant adeno-associated virus-mediated high-efficiency, transient expression of the murine cationic amino acid transporter (Ecotropic retroviral receptor) permits stable transduction of human HeLa cells by ecotropic retroviral vectors [J].
Bertran, J ;
Miller, JL ;
Yang, YP ;
FenimoreJustman, A ;
Rueda, F ;
Vanin, EF ;
Nienhuis, AW .
JOURNAL OF VIROLOGY, 1996, 70 (10) :6759-6766
[5]   Stimulation of homologous recombination through targeted cleavage by chimeric nucleases [J].
Bibikova, M ;
Carroll, D ;
Segal, DJ ;
Trautman, JK ;
Smith, J ;
Kim, YG ;
Chandrasegaran, S .
MOLECULAR AND CELLULAR BIOLOGY, 2001, 21 (01) :289-297
[6]   Enhancing gene targeting with designed zinc finger nucleases [J].
Bibikova, M ;
Beumer, K ;
Trautman, JK ;
Carroll, D .
SCIENCE, 2003, 300 (5620) :764-764
[7]   Stimulation of intrachromosomal homologous recombination in human cells by electroporation with site-specific endonucleases [J].
Brenneman, M ;
Gimble, FS ;
Wilson, JH .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1996, 93 (08) :3608-3612
[8]  
BROWN PO, 1990, CURR TOP MICROBIOL, V157, P19
[9]   Gene targeting in stem cells from individuals with osteogenesis imperfecta [J].
Chamberlain, JR ;
Schwarze, U ;
Wang, PR ;
Hirata, RK ;
Hankenson, KD ;
Pace, JM ;
Underwood, RA ;
Song, KM ;
Sussman, M ;
Byers, PH ;
Russell, DW .
SCIENCE, 2004, 303 (5661) :1198-1201
[10]   Expression of human factor VIII by splicing between dimerized AAV vectors [J].
Chao, HJ ;
Sun, LW ;
Bruce, A ;
Xiao, X ;
Walsh, CE .
MOLECULAR THERAPY, 2002, 5 (06) :716-722