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, β-thalassemia, and Duchenne muscular dystrophy, suggesting that SFHR has potential as a therapeutic modality for the treatment of monogenic inherited disease.
Authors
Dieter C. Gruenert, Emanuela Bruscia, Giuseppe Novelli, Alessia Colosimo, Bruno Dallapiccola, Federica Sangiuolo, Kaarin K. Goncz
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