CRISPR/Cas9-mediated A4GALT suppression rescues Fabry disease phenotypes in a kidney organoid model

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 A4GALT KO in both CMC-Fb-002 and GLA-KO to make Fb-002-A4GALT-KO and GLA/A4GALT-KO, respectively.

Source: Translational Research - February 17, 2023 Category: Research Authors: Sheng Cui, Yoo Jin Shin, Xianying Fang, Hanbi Lee, Sang Hun Eum, Eun Jeong Ko, Sun Woo Lim, Eunji Shin, Kang In Lee, Jae Young Lee, Chae Bin Lee, Soo Kyung Bae, Chul Woo Yang, Byung Ha Chung Source Type: research