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.
Gruenert
Dieter C
DC
Department of Medicine, University of Vermont, Burlington, Vermont, USA. dieter@cooper.cpmc.org
Bruscia
Emanuela
E
Novelli
Giuseppe
G
Colosimo
Alessia
A
Dallapiccola
Bruno
B
Sangiuolo
Federica
F
Goncz
Kaarin K
KK
D000755:Anemia, Sickle Cell / Q000628:therapy; D000818:Animals; D001483:Base Sequence; D003550:Cystic Fibrosis / Q000628:therapy; D004247:DNA / Q000627:therapeutic use*; D018390:Gene Targeting; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000628:therapy
D004247:DNA