Department of Pharmacology, Central University of Punjab, Ghudda, Bathinda, Punjab, 151401, India.
Pharmacol Rep. 2024 Aug;76(4):693-713. doi: 10.1007/s43440-024-00619-z. Epub 2024 Jul 9.
Huntington's disease (HD) is an inherited neurodegenerative disorder caused by an expansion in CAG repeat on huntington (Htt) gene, leading to a degeneration of GABAergic medium spiny neurons (MSNs) in the striatum, resulting in the generation of reactive oxygen species, and decrease antioxidant activity. These pathophysiological alterations impair mitochondrial functions, leading to an increase in involuntary hyperkinetic movement. However, researchers investigated the neuroprotective effect of antioxidants using various animal models. Still, their impact is strictly limited to curtailing oxidative stress and increasing the antioxidant enzyme in the brain, which is less effective in HD. Meanwhile, researchers discovered Mitochondria-targeted antioxidants (MTAXs) that can improve mitochondrial functions and antioxidant activity through the modulation of mitochondrial signaling pathways, including peroxisome proliferator-activated receptor (PPAR)-coactivator 1 (PGC-1α), dynamin-related protein 1 (Drp1), mitochondrial fission protein 1 (Fis1), and Silent mating type information regulation 2 homolog 1 (SIRT-1), showing neuroprotective effects in HD. The present review discusses the clinical and preclinical studies that investigate the neuroprotective effect of MTAXs (SS31, XJB-5-131, MitoQ, bezafibrate, rosiglitazone, meldonium, coenzyme Q10, etc.) in HD. This brief literature review will help to understand the relevance of MTAXs in HD and enlighten the importance of MTAXs in future drug discovery and development.
亨廷顿病 (HD) 是一种遗传性神经退行性疾病,由亨廷顿基因 (Htt) 上 CAG 重复扩增引起,导致纹状体 GABA 能中间神经元 (MSNs) 退化,产生活性氧,降低抗氧化活性。这些病理生理改变损害线粒体功能,导致不自主的运动过度。然而,研究人员使用各种动物模型研究了抗氧化剂的神经保护作用。尽管如此,它们的影响仅限于减轻氧化应激和增加大脑中的抗氧化酶,在 HD 中效果较差。同时,研究人员发现线粒体靶向抗氧化剂 (MTAXs) 可以通过调节线粒体信号通路,包括过氧化物酶体增殖物激活受体 (PPAR)-共激活因子 1 (PGC-1α)、动力相关蛋白 1 (Drp1)、线粒体分裂蛋白 1 (Fis1) 和沉默交配型信息调节 2 同源物 1 (SIRT-1),改善线粒体功能和抗氧化活性,从而发挥 HD 的神经保护作用。本综述讨论了研究 MTAXs (SS31、XJB-5-131、MitoQ、贝扎贝特、罗格列酮、美托洛尔、辅酶 Q10 等) 在 HD 中神经保护作用的临床前和临床研究。这篇简短的文献综述将有助于理解 MTAXs 在 HD 中的相关性,并阐明 MTAXs 在未来药物发现和开发中的重要性。