title: Sequence-specific modification of genomic DNA by small DNA fragments.
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.
authors.affiliation: Department of Medicine, University of Vermont, Burlington, Vermont, USA. dieter@cooper.cpmc.org
authors.forename: Dieter C
authors.initials: DC
authors.lastname: Gruenert
authors.forename: Emanuela
authors.initials: E
authors.lastname: Bruscia
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Novelli
authors.forename: Alessia
authors.initials: A
authors.lastname: Colosimo
authors.forename: Bruno
authors.initials: B
authors.lastname: Dallapiccola
authors.forename: Federica
authors.initials: F
authors.lastname: Sangiuolo
authors.forename: Kaarin K
authors.initials: KK
authors.lastname: Goncz
mesh_terms: 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
chemical_list: D004247:DNA