Translational Research Institute of Brain and Brain-like Intelligence, Shanghai Key Laboratory of Anesthesiology and Brain Functional Modulation, Department of Pediatrics, Shanghai Fourth People's Hospital, School of Medicine, Tongji University, Shanghai 200434, China; Department of Medical Genetics, Tongji University School of Medicine, Shanghai 200092, China; Department of Ophthalmology, Tongji Hospital, School of Medicine, Tongji University, Shanghai 200065, China.
Translational Research Institute of Brain and Brain-like Intelligence, Shanghai Key Laboratory of Anesthesiology and Brain Functional Modulation, Department of Pediatrics, Shanghai Fourth People's Hospital, School of Medicine, Tongji University, Shanghai 200434, China; Department of Medical Genetics, Tongji University School of Medicine, Shanghai 200092, China.
Toxicology. 2023 Jul;493:153555. doi: 10.1016/j.tox.2023.153555. Epub 2023 May 24.
Cysteamine, a sulfhydryl compound, is an intermediate in the metabolism of coenzyme A to taurine in living organisms. However, the potential side effects of cysteamine such as hepatotoxicity in pediatric patients have been reported in some studies. To evaluate the impact of cysteamine on infants and children, larval zebrafish (a vertebrate model) were exposed to 0.18, 0.36 and 0.54 mM cysteamine from 72 hpf to 144 hpf. Alterations in general and pathological evaluation, biochemical parameters, cell proliferation, lipid metabolism factors, inflammatory factors and Wnt signaling pathway levels were examined. Increased liver area and lipid accumulation were observed in liver morphology, staining and histopathology in a dose-dependent manner with cysteamine exposure. In addition, the experimental cysteamine group exhibited higher alanine aminotransferase, aspartate aminotransferase, total triglyceride and total cholesterol levels than the control group. Meanwhile, the levels of lipogenesis-related factors ascended whereas lipid transport-related factors descended. Oxidative stress indicators such as reactive oxygen species, MDA and SOD were upregulated after cysteamine exposure. Afterwards, transcription assays revealed that biotinidase and Wnt pathway-related genes were upregulated in the exposed group, and inhibition of Wnt signaling partially rescued the abnormal liver development. The current study found that cysteamine-induced hepatotoxicity in larval zebrafish is due to inflammation and abnormal lipid metabolism, which is mediated by biotinidase (a potential pantetheinase isoenzyme) and Wnt signaling. This provides a perspective on the safety of cysteamine administration in children and identifies potential targets for protection against adverse reactions.
半胱胺是一种巯基化合物,是生物体内辅酶 A 代谢为牛磺酸的中间产物。然而,一些研究报道了半胱胺在儿科患者中的潜在副作用,如肝毒性。为了评估半胱胺对婴儿和儿童的影响,从 72 hpf 到 144 hpf,将幼虫斑马鱼(一种脊椎动物模型)暴露于 0.18、0.36 和 0.54 mM 的半胱胺中。检查了一般和病理评估、生化参数、细胞增殖、脂质代谢因子、炎症因子和 Wnt 信号通路水平的变化。随着半胱胺暴露,肝脏形态、染色和组织病理学观察到肝区面积增加和脂质积累呈剂量依赖性。此外,实验组丙氨酸氨基转移酶、天冬氨酸氨基转移酶、总甘油三酯和总胆固醇水平均高于对照组。同时,与脂质生成相关的因子水平上升,而与脂质转运相关的因子水平下降。暴露于半胱胺后,氧化应激指标如活性氧、MDA 和 SOD 上调。随后,转录分析显示暴露组中生物素酶和 Wnt 途径相关基因上调,而 Wnt 信号通路的抑制部分挽救了异常的肝脏发育。本研究发现,半胱胺诱导的幼虫斑马鱼肝毒性是由于炎症和异常的脂质代谢,这是由生物素酶(一种潜在的泛肽酶同工酶)和 Wnt 信号介导的。这为儿童半胱胺给药的安全性提供了新视角,并确定了潜在的保护目标,以防止不良反应。