Article
CRISPR/Cas9-mediated A4GALT suppression rescues Fabry disease phenotypes in a kidney organoid model.
Translational research : the journal of laboratory and clinical medicine - 1 Aug 2023
Cui Sheng, Shin Yoo Jin, Fang Xianying, Lee Hanbi, Eum Sang Hun, Ko Eun Jeong, Lim Sun Woo, Shin Eunji, Lee Kang In, Lee Jae Young, Lee Chae Bin, Bae Soo Kyung, Yang Chul Woo, Chung Byung Ha
Abstract excerpt
The objective of this study was to investigate whether CRISPR/Cas9-mediated suppression of A4GALT could rescue phenotype of Fabry disease nephropathy (FDN) using human induced pluripotent stem cells (hiPSCs) derived kidney organoid system. We generated FDN patient-derived hiPSC (CMC-Fb-002) and FD-specific hiPSCs (GLA-KO) by knock-out (KO) of GLA in wild-type (WT) hiPSCs using CRISPR/Cas9. We then performed...
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