Department of Pharmacology, School of Pharmacy, Fudan University, 826 Zhangheng Road, Shanghai 201203, China.
Center for Drug Safety Evaluation and Research, State Key Laboratory of Drug Research, Shanghai Institute of Materia Medica, Chinese Academy of Sciences, 501 Haike Road, Shanghai 201203, China.
Int J Mol Sci. 2022 Dec 14;23(24):15931. doi: 10.3390/ijms232415931.
Non-alcoholic fatty liver disease (NAFLD) is a chronic metabolic disease manifested in hepatic steatosis, inflammation, fibrosis, etc., which affects over one-quarter of the population around the world. Since no effective therapeutic drugs are available to cope with this widespread epidemic, the functional research of genes with altered expression during NAFLD helps understand the pathogenesis of this disease and the development of new potential therapeutic targets for drugs. In the current work, we discovered via the analysis of the Gene Expression Omnibus (GEO) dataset that cysteine sulfinic acid decarboxylase (CSAD) decreased significantly in NAFLD patients, which was also confirmed in multiple NAFLD mouse models (HFD-fed C57BL/6J, db/db and HFHFrHC-fed C57BL/6J mice). Next, CSAD's function in the progression of NAFLD was explored using AAV-mediated liver-directed gene overexpression in an HFD-fed mouse model, where the overexpression of CSAD in the liver could alleviate NAFLD-associated pathologies, including body weight, liver/body weight ratio, hepatic triglyceride and total cholesterol, and the degree of steatosis. Mechanically, we found that the overexpression of CSAD could increase the expression of some genes related to fatty acid β-oxidation (Acad1, Ppara, and Acox1). Furthermore, we also detected that CSAD could improve mitochondrial injury in vitro and in vivo. Finally, we proposed that the effect of CSAD on lipid accumulation might be independent of the taurine pathway. In conclusion, we demonstrated that CSAD is involved in the development of NAFLD as a protective factor, which suggested that CSAD has the potential to become a new target for drug discovery in NAFLD.
非酒精性脂肪性肝病 (NAFLD) 是一种以肝脂肪变性、炎症、纤维化等为特征的慢性代谢性疾病,影响着全球超过四分之一的人口。由于目前尚无有效的治疗药物来应对这种广泛流行的疾病,因此研究 NAFLD 时基因表达改变的功能有助于了解该疾病的发病机制,并为药物开发新的潜在治疗靶点提供依据。在本研究中,我们通过对基因表达综合数据库 (GEO) 数据集的分析发现,胱硫醚脱羧酶 (CSAD) 在 NAFLD 患者中显著下调,这一结果在多个 NAFLD 小鼠模型 (高脂饮食喂养的 C57BL/6J、db/db 和 HFHFrHC 喂养的 C57BL/6J 小鼠) 中也得到了验证。接下来,我们通过在高脂饮食喂养的小鼠模型中使用 AAV 介导的肝脏定向基因过表达来研究 CSAD 在 NAFLD 进展中的作用,结果发现肝脏中 CSAD 的过表达可以缓解与 NAFLD 相关的病理变化,包括体重、肝重/体重比、肝内甘油三酯和总胆固醇以及脂肪变性程度。从机制上看,我们发现 CSAD 的过表达可以增加一些与脂肪酸 β-氧化相关的基因的表达 (Acad1、Ppara 和 Acox1)。此外,我们还检测到 CSAD 可以改善体外和体内的线粒体损伤。最后,我们提出 CSAD 对脂质积累的影响可能独立于牛磺酸途径。总之,我们证明 CSAD 作为一种保护因子参与了 NAFLD 的发生发展,这提示 CSAD 有可能成为 NAFLD 药物治疗的新靶点。