Ontology highlight
ABSTRACT:
SUBMITTER: Osborn M
PROVIDER: S-EPMC5035838 | biostudies-other | 2016 Aug
REPOSITORIES: biostudies-other
Osborn Mark M Lonetree Cara-Lin CL Webber Beau R BR Patel Dharmeshkumar D Dunmire Samantha S McElroy Amber N AN DeFeo Anthony P AP MacMillan Margaret L ML Wagner John J Balzar Bruce R BR Tolar Jakub J
Stem cells and development 20160818 20
The ability to rationally target disease-causing mutations has been made possible with programmable nucleases with the CRISPR/Cas9 system representing a facile platform for individualized gene-based medicine. In this study we employed footprint free reprogramming of fibroblasts from a patient with mutations to the Fanconi anemia I (FANCI) gene to generate induced pluripotent stem cells (iPSC). This process was accomplished without gene complementation and the resultant iPSC were able to be gene ...[more]