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J Clin Invest. 2003 Sep;112(5):637-41.

Sequence-specific modification of genomic DNA by small DNA fragments.

Author information

1
Department of Medicine, University of Vermont, Burlington, Vermont, USA. dieter@cooper.cpmc.org

Abstract

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.

PMID:
12952908
PMCID:
PMC182219
DOI:
10.1172/JCI19773
[Indexed for MEDLINE]
Free PMC Article

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