Research Biomarkers, Amgen Research, South San Francisco, California, USA.
Cardiometabolic Disorders, Amgen Research, South San Francisco, California, USA.
Hepatol Commun. 2024 May 2;8(5). doi: 10.1097/HC9.0000000000000419. eCollection 2024 May 1.
Human genetic studies have identified several mitochondrial amidoxime-reducing component 1 (MTARC1) variants as protective against metabolic dysfunction-associated steatotic liver disease. The MTARC1 variants are associated with decreased plasma lipids and liver enzymes and reduced liver-related mortality. However, the role of mARC1 in fatty liver disease is still unclear.
Given that mARC1 is mainly expressed in hepatocytes, we developed an N-acetylgalactosamine-conjugated mouse Mtarc1 siRNA, applying it in multiple in vivo models to investigate the role of mARC1 using multiomic techniques.
In ob/ob mice, knockdown of Mtarc1 in mouse hepatocytes resulted in decreased serum liver enzymes, LDL-cholesterol, and liver triglycerides. Reduction of mARC1 also reduced liver weight, improved lipid profiles, and attenuated liver pathological changes in 2 diet-induced metabolic dysfunction-associated steatohepatitis mouse models. A comprehensive analysis of mARC1-deficient liver from a metabolic dysfunction-associated steatohepatitis mouse model by metabolomics, proteomics, and lipidomics showed that Mtarc1 knockdown partially restored metabolites and lipids altered by diet.
Taken together, reducing mARC1 expression in hepatocytes protects against metabolic dysfunction-associated steatohepatitis in multiple murine models, suggesting a potential therapeutic approach for this chronic liver disease.
人类遗传研究已经确定了几种线粒体氨肟还原酶 1(MTARC1)变体作为代谢功能障碍相关脂肪性肝病的保护因子。MTARC1 变体与降低的血浆脂质和肝酶以及降低的与肝相关的死亡率相关。然而,mARC1 在脂肪性肝病中的作用仍不清楚。
鉴于 mARC1 主要在肝细胞中表达,我们开发了一种 N-乙酰半乳糖胺缀合的小鼠 Mtarc1 siRNA,并应用于多种体内模型,使用多组学技术来研究 mARC1 的作用。
在 ob/ob 小鼠中,敲低小鼠肝细胞中的 Mtarc1 导致血清肝酶、LDL-胆固醇和肝甘油三酯降低。mARC1 的减少还降低了肝重、改善了脂质谱,并减轻了 2 种饮食诱导的代谢功能障碍相关脂肪性肝炎小鼠模型中的肝病理变化。通过代谢组学、蛋白质组学和脂质组学对代谢功能障碍相关脂肪性肝炎小鼠模型中 mARC1 缺陷的肝脏进行综合分析表明,Mtarc1 敲低部分恢复了饮食改变的代谢物和脂质。
总之,降低肝细胞中的 mARC1 表达可预防多种小鼠模型中的代谢功能障碍相关脂肪性肝炎,提示这种慢性肝病的潜在治疗方法。