Sequence-specific modification of genomic DNA by small DNA fragments

被引：57

作者：

Gruenert, DC

Bruscia, E

Novelli, G

Colosimo, A

Dallapiccola, B

Sangiuolo, F

Goncz, KK

机构：

[1] Univ Vermont, Dept Med, Human Mol Genet Unit, Burlington, VT USA

[2] Univ Roma Tor Vergata, Dept Biopathol & Diagnost Imaging, Rome, Italy

[3] CSS Mendel Inst, Rome, Italy

[4] Univ G dAnnunzio, Dept Biomed Sci, Chieti, Italy

[5] Univ Roma La Sapienza, Dept Expt Med & Pathol, Rome, Italy

来源：

JOURNAL OF CLINICAL INVESTIGATION | 2003年 / 112卷 / 05期

关键词：

D O I：

10.1172/JCI200319773

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

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.

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页码：637 / 641

页数：5

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