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Review
. 2003 Sep;112(5):637-41.
doi: 10.1172/JCI19773.

Sequence-specific modification of genomic DNA by small DNA fragments

Dieter C Gruenert  1 , Emanuela Bruscia, Giuseppe Novelli, Alessia Colosimo, Bruno Dallapiccola, Federica Sangiuolo, Kaarin K Goncz
Affiliations
Review

Sequence-specific modification of genomic DNA by small DNA fragments

Dieter C Gruenert et al. J Clin Invest. 2003 Sep.

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.

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Figures

Figure 1
Figure 1
Schematic representation of different SDF configurations possible in SFHR-mediated modification of a target sequence. The SDF can interact with the target sequence as (a) sense and/or (b) antisense ssDNA, or as (c) dsDNA. The ssDNA can be introduced either as individual strands (sense and antisense) or as denatured cDNA strands. The enzymatic mechanisms involved require further elucidation.
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