The development and regulation of gene repair

被引:54
作者
Liu, L [1 ]
Parekh-Olmedo, H [1 ]
Kmiec, EB [1 ]
机构
[1] Univ Delaware, Dept Biol Sci, Newark, DE 19716 USA
基金
美国国家卫生研究院;
关键词
D O I
10.1038/nrg1156
中图分类号
Q3 [遗传学];
学科分类号
071007 ; 090102 ;
摘要
0A technique that can direct the repair of a genetic mutation in a human chromosome using the DNA repair machinery of the cell is under development. Although this approach is not as mature as other forms of gene therapy and fundamental problems continue to arise, it promises to be the ultimate therapy for many inherited disorders. There is a continuing effort to understand the potential and the limitations of this controversial approach.
引用
收藏
页码:679 / 689
页数:11
相关论文
共 104 条
[41]   Targeted gene correction by small single-stranded oligonucleotides in mammalian cells [J].
Igoucheva, O ;
Alexeev, V ;
Yoon, K .
GENE THERAPY, 2001, 8 (05) :391-399
[42]   Transcription affects formation and processing of intermediates in oligonucleotide-mediated gene alteration [J].
Igoucheva, O ;
Alexeev, V ;
Pryce, M ;
Yoon, K .
NUCLEIC ACIDS RESEARCH, 2003, 31 (10) :2659-2670
[43]   A sequence-specific gene correction by an RNA-DNA oligonucleotide in mammalian cells characterized by transfection and nuclear extract using a lacZ shuttle system [J].
Igoucheva, O ;
Peritz, AE ;
Levy, D ;
Yoon, K .
GENE THERAPY, 1999, 6 (12) :1960-1971
[44]   Targeted single-base correction by RNA-DNA oligonucleotides [J].
Igoucheva, O ;
Yoon, K .
HUMAN GENE THERAPY, 2000, 11 (16) :2307-2312
[45]   COMPLEMENT-STABILIZED D-LOOP - RECA-CATALYZED STABLE PAIRING OF LINEAR DNA-MOLECULES AT INTERNAL SITES [J].
JAYASENA, VK ;
JOHNSTON, BH .
JOURNAL OF MOLECULAR BIOLOGY, 1993, 230 (03) :1015-1024
[46]   In vivo and in vitro correction of the mdx dystrophin gene nonsense mutation by short-fragment homologous replacement [J].
Kapsa, R ;
Quigley, A ;
Lynch, GS ;
Steeper, K ;
Kornberg, AJ ;
Gregorevic, P ;
Austin, L ;
Byrne, E .
HUMAN GENE THERAPY, 2001, 12 (06) :629-642
[47]   Chloroplast lysates support directed mutagenesis via modified DNA and chimeric RNA/DNA oligonucleotides [J].
Kmiec, EB ;
Johnson, C ;
May, GD .
PLANT JOURNAL, 2001, 27 (03) :267-274
[48]   Targeted gene repair [J].
Kmiec, EB .
GENE THERAPY, 1999, 6 (01) :1-3
[49]  
KMIEC EB, 1998, DEV HUMAN GENE THERA, P101
[50]   Chimeric RNA/DNA oligonucleotide-based site-specific modification of the tobacco acetolactate syntase gene [J].
Kochevenko, A ;
Willmitzer, L .
PLANT PHYSIOLOGY, 2003, 132 (01) :174-184