Sequence-specific modification of genomic DNA by small DNA fragments.
Sequence-specific modification of genomic DNA by small DNA fragments.
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通过小 DNA 片段对基因组 DNA 进行序列特异性修饰。
DOI：
10.1172/jci19773
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发表时间：
2003
期刊：
影响因子：
--
通讯作者：
K. Goncz
中科院分区：
文献类型：
--
作者：
D. Gruenert;E. Bruscia;G. Novelli;A. Colosimo;B. Dallapiccola;F. Sangiuolo;K. Goncz
在人类和小鼠细胞中，小DNA片段被用来修饰内源基因组DNA。这种被称为小片段同源替换(SFHR)的序列特异性修饰或基因组编辑策略，尚未就其潜在机制进行表征。SFHR后的基因分型和表型分析显示，与囊性纤维化、β-地中海贫血和Duchenne肌营养不良相关的致病基因位点发生了特异性的修饰，这表明SFHR有可能成为治疗单基因遗传病的一种方法。
Small DNA fragments have been used to modify endogenous genomic DNA in both human and mouse cells. This strategy for sequence-specific modification or genomic editing, known as small-fragment homologous replacement (SFHR), has yet to be characterized in terms of its underlying mechanisms. Genotypic and phenotypic analyses following SFHR have shown specific modification of disease-causing genetic loci associated with cystic fibrosis, beta-thalassemia, and Duchenne muscular dystrophy, suggesting that SFHR has potential as a therapeutic modality for the treatment of monogenic inherited disease.
影响因子：
56.9
作者：
Vasquez, KM;Narayanan, L;Glazer, PM
通讯作者：
Glazer, PM
同源重组涉及人类细胞中的小单链寡核苷酸。
DOI：
--
发表时间：
1989
期刊：
The New biologist
影响因子：
--
作者：
Campbell,CR;Keown,W;Lowe,L;Kirschling,D;Kucherlapati,R
通讯作者：
Kucherlapati,R