Section of Integrative Physiology and Metabolism, Joslin Diabetes Center, Harvard Medical School, Boston, MA, USA.
Alnylam Pharmaceuticals, Cambridge, MA, USA.
J Lipid Res. 2023 Feb;64(2):100324. doi: 10.1016/j.jlr.2022.100324. Epub 2022 Dec 29.
Nonalcoholic fatty liver disease (NAFLD) is the most common chronic liver disease with increased risk in patients with metabolic syndrome. There are no FDA-approved treatments, but FXR agonists have shown promising results in clinical studies for NAFLD management. In addition to FXR, fibroblast growth factor receptor FGFR4 is a key mediator of hepatic bile acid synthesis. Using N-acetylgalactosamine-conjugated siRNA, we knocked down FGFR4 specifically in the liver of mice on chow or high-fat diet and in mouse primary hepatocytes to determine the role of FGFR4 in metabolic processes and hepatic steatosis. Liver-specific FGFR4 silencing increased bile acid production and lowered serum cholesterol. Additionally, we found that high-fat diet-induced liver steatosis and insulin resistance improved following FGFR4 knockdown. These improvements were associated with activation of the FXR-FGF15 axis in intestinal cells, but not in hepatocytes. We conclude that targeting FGFR4 in the liver to activate the intestinal FXR-FGF15 axis may be a promising strategy for the treatment of NAFLD and metabolic dysfunction.
非酒精性脂肪性肝病(NAFLD)是最常见的慢性肝病,代谢综合征患者的风险增加。目前还没有获得 FDA 批准的治疗方法,但 FXR 激动剂在 NAFLD 管理的临床研究中显示出有希望的结果。除了 FXR,成纤维细胞生长因子受体 FGFR4 是肝脏胆汁酸合成的关键介质。我们使用 N-乙酰半乳糖胺缀合的 siRNA,在正常饮食或高脂肪饮食的小鼠和小鼠原代肝细胞中特异性敲低 FGFR4,以确定 FGFR4 在代谢过程和肝脂肪变性中的作用。肝特异性 FGFR4 沉默增加了胆汁酸的产生并降低了血清胆固醇。此外,我们发现 FGFR4 敲低后,高脂肪饮食诱导的肝脂肪变性和胰岛素抵抗得到改善。这些改善与肠细胞中 FXR-FGF15 轴的激活有关,但与肝细胞无关。我们得出结论,靶向肝脏中的 FGFR4 以激活肠道 FXR-FGF15 轴可能是治疗 NAFLD 和代谢功能障碍的有前途的策略。