Article
Blood vessel organoids generated by base editing and harboring single nucleotide variation in Notch3 effectively recapitulate CADASIL-related pathogenesis.
Molecular neurobiology - 1 Nov 2024
Ahn Yujin, An Ju-Hyun, Yang Hae-Jun, Lee Wi-Jae, Lee Sang-Hee, Park Young-Ho, Lee Jong-Hee, Lee Hong J, Lee Seung Hwan, Kim Sun-Uk
Abstract excerpt
Human blood vessel organoids (hBVOs) offer a promising platform for investigating vascular diseases and identifying therapeutic targets. In this study, we focused on in vitro modeling and therapeutic target finding of cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL), the most common form of hereditary stroke disorder caused by mutations in the NOTCH3 gene....
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