Generation and characterization of the human iPSC line IDISi001-A isolated from blood cells of a CADASIL patient carrying a NOTCH3 mutation

Human iPSCs generated from CADASIL patients will A) facilitate studies of developmental biology of endothelial cells and vascular smooth muscular cells pathologically disrupted in CADASIL; B) yield endothelial and vascular smooth muscular cells for in vitro CADASIL model for drug screening and C) help to establish the therapeutic utility of iPSCs corrected by CRISPR-Cas9 in animal models of CADASIL (e.g., in Notch3−/− mice).167