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

Yoo Jin ShinSeung Yun ChaeHanbi LeeXianying FangSheng CuiSun Woo LimKang In LeeJae Young LeeCan LiChul Woo YangByung Ha Chung

Published in: Atherosclerosis (2024)

CRISPR/Cas9-mediated A4GALT suppression rescued FD phenotype and endothelial dysfunction in GLA-mutant hiPSC-ECs, presenting a potential therapeutic approach for FD-vasculopathy.

Keyphrases

induced pluripotent stem cells
crispr cas
genome editing
endothelial cells
oxidative stress
mouse model
replacement therapy
high glucose
left ventricular
hypertrophic cardiomyopathy