Ontology highlight
ABSTRACT:
SUBMITTER: Laskowski TJ
PROVIDER: S-EPMC4982969 | biostudies-literature | 2016 Aug
REPOSITORIES: biostudies-literature
Laskowski Tamara J TJ Van Caeneghem Yasmine Y Pourebrahim Rasoul R Ma Chao C Ni Zhenya Z Garate Zita Z Crane Ana M AM Li Xuan Shirley XS Liao Wei W Gonzalez-Garay Manuel M Segovia Jose Carlos JC Paschon David E DE Rebar Edward J EJ Holmes Michael C MC Kaufman Dan D Vandekerckhove Bart B Davis Brian R BR
Stem cell reports 20160707 2
Wiskott-Aldrich syndrome (WAS) is an X-linked primary immunodeficiency disease caused by mutations in the gene encoding the WAS protein (WASp). Here, induced pluripotent stem cells (iPSCs) were derived from a WAS patient (WAS-iPSC) and the endogenous chromosomal WAS locus was targeted with a wtWAS-2A-eGFP transgene using zinc finger nucleases (ZFNs) to generate corrected WAS-iPSC (cWAS-iPSC). WASp and GFP were first expressed in the earliest CD34(+)CD43(+)CD45(-) hematopoietic precursor cells an ...[more]