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CRISPR/Cas9-mediated suppression of A4GALT rescues endothelial cell dysfunction in a fabry disease vasculopathy model derived from human induced pluripotent stem cells.

Atherosclerosis - 1 Oct 2024

Shin Yoo Jin, Chae Seung Yun, Lee Hanbi, Fang Xianying, Cui Sheng, Lim Sun Woo, Lee Kang In, Lee Jae Young, Li Can, Yang Chul Woo, Chung Byung Ha

Abstract excerpt

BACKGROUND AND AIMS: The objective of this study was to investigate the efficacy of CRISPR/Cas9-mediated A4GALT suppression in rescuing endothelial dysfunction in Fabry disease (FD) endothelial cells (FD-ECs) derived from human induced pluripotent stem cells (hiPSCs). METHODS: We differentiated hiPSCs (WT (wild-type), WTC-11), GLA-mutant hiPSCs (GLA-KO, CMC-Fb-002), and CRISPR/Cas9-mediated A4GALT-KO hiPSCs...

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PMID: 39141976
DOI: 10.1016/j.atherosclerosis.2024.118549

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