Correction of a CADASIL Point Mutation Using Adenine Base Editors in hiPSCs and Blood Vessel Organoids

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Vascular smooth muscle cells (VSMCs) differentiated from cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL) human induced pluripotent stem cells (hiPSCs) showed NOTCH3 deposition and abnormal actin cytoskeleton structure, and the abnormalities were recovered in corrected hiPSC-derived VSMCs.
[Journal Of Genetics And Genomics]
Abstract