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.
Gruenert, D., Bruscia, E., Novelli, G., Colosimo, A., Dallapiccola, B., Sangiuolo, F.c., et al. (2003). Sequence-specific modification of genomic DNA by small DNA fragments. THE JOURNAL OF CLINICAL INVESTIGATION, 112(5), 637-641 [10.1172/JCI19773].
Sequence-specific modification of genomic DNA by small DNA fragments
NOVELLI, GIUSEPPE;SANGIUOLO, FEDERICA CARLA;
2003-09-01
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.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
