Ontology highlight
ABSTRACT:
SUBMITTER: Osborn MJ
PROVIDER: S-EPMC4326027 | biostudies-literature | 2015 Feb
REPOSITORIES: biostudies-literature
Osborn Mark J MJ Gabriel Richard R Webber Beau R BR DeFeo Anthony P AP McElroy Amber N AN Jarjour Jordan J Starker Colby G CG Wagner John E JE Joung J Keith JK Voytas Daniel F DF von Kalle Christof C Schmidt Manfred M Blazar Bruce R BR Tolar Jakub J
Human gene therapy 20150201 2
Genome engineering with designer nucleases is a rapidly progressing field, and the ability to correct human gene mutations in situ is highly desirable. We employed fibroblasts derived from a patient with Fanconi anemia as a model to test the ability of the clustered regularly interspaced short palindromic repeats/Cas9 nuclease system to mediate gene correction. We show that the Cas9 nuclease and nickase each resulted in gene correction, but the nickase, because of its ability to preferentially m ...[more]