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Hoffmann, R. A wiki for the life sciences where authorship matters. Nature Genetics (2008)

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

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.[1]


  1. Sequence-specific modification of genomic DNA by small DNA fragments. Gruenert, D.C., Bruscia, E., Novelli, G., Colosimo, A., Dallapiccola, B., Sangiuolo, F., Goncz, K.K. J. Clin. Invest. (2003) [Pubmed]
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