Ontology highlight
ABSTRACT:
SUBMITTER: Jiang J
PROVIDER: S-EPMC3848249 | biostudies-literature | 2013 Aug
REPOSITORIES: biostudies-literature
Nature 20130717 7462
Down's syndrome is a common disorder with enormous medical and social costs, caused by trisomy for chromosome 21. We tested the concept that gene imbalance across an extra chromosome can be de facto corrected by manipulating a single gene, XIST (the X-inactivation gene). Using genome editing with zinc finger nucleases, we inserted a large, inducible XIST transgene into the DYRK1A locus on chromosome 21, in Down's syndrome pluripotent stem cells. The XIST non-coding RNA coats chromosome 21 and tr ...[more]