Small molecule APOL1 inhibitors as a precision medicine approach for APOL1-mediated kidney disease

• Published in: Nature communications Vol. 16; no. 1; pp. 167 - 13
• Main Authors: Zimmerman, Brandon, Dakin, Leslie A., Fortier, Anne, Nanou, Evanthia, Blasio, Angelo, Mann, James, Miller, Howard, Fletcher, Marissa, Wang, Tiansheng, Nanthakumar, Suganthini, McCarthy, Gizelle, Matar, Caline, Matsye, Prachi, Wang, Guanyu, Snyder, Phillip, Daniel, Kevin, Swamy, Harsha, Sullivan, Kelly, Bright, Franklin, Powers, Audrey, Gagnon, Kevin J., Lu, Fan, Paula, Steven, Khare-Pandit, Suvarna, Henry, Larry, Hamel, Martine, Denis, Francois, Nicolas, Olivier, Hariparsad, Niresh, Kumar, Shyamesh, Proctor, Jennifer, Senter, Timothy, Furey, Brinley, Bunnage, Mark E.
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 02.01.2025; Nature Publishing Group; Nature Portfolio
• Subjects: 13/1; 13/31; 13/51; 140/131; 38/47; 631/154/556; 64/110; 692/4022/1585/104/1586; 692/4022/1585/2759/1523; 82/80; 82/83; 9/74; 96/21; 96/98; Animals; Apolipoprotein L1 - genetics; Apolipoprotein L1 - metabolism; Apolipoproteins; Cell death; Channel gating; Disease Models, Animal; Female; Genetic diversity; Genetic variance; Genome-wide association studies; Health services; Humanities and Social Sciences; Humans; Inhibitors; Ion channels; Kidney diseases; Kidneys; Male; Medical treatment; Mice; Mice, Transgenic; multidisciplinary; Podocytes - drug effects; Podocytes - metabolism; Podocytes - pathology; Precision medicine; Precision Medicine - methods; Renal Insufficiency, Chronic - drug therapy; Renal Insufficiency, Chronic - genetics; Renal Insufficiency, Chronic - metabolism; Renal Insufficiency, Chronic - pathology; Science; Science (multidisciplinary); Transgenic mice

Chronic kidney disease affects ~10% of people worldwide and there are no disease modifying therapeutics that address the underlying cause of any form of kidney disease. Genome wide association studies have identified the G1 and G2 variants in the apolipoprotein L1 ( APOL1 ) gene as major contributor...