Centro de Investigaciones Biológicas-CSIC, Ramiro de Maeztu 9, 28040 Madrid, Spain.
Centro de Investigación Biomédica en Red de Enfermedades Neurodegenerativas (CIBERNED), Instituto de Salud Carlos III, 28031 Madrid, Spain.
Int J Mol Sci. 2021 Jan 13;22(2):740. doi: 10.3390/ijms22020740.
Amyotrophic lateral sclerosis (ALS) is a lethal neurodegenerative disease that usually results in respiratory paralysis in an interval of 2 to 4 years. ALS shows a multifactorial pathogenesis with an unknown etiology, and currently lacks an effective treatment. The vast majority of patients exhibit protein aggregation and a dysfunctional mitochondrial accumulation in their motoneurons. As a result, autophagy and mitophagy modulators may be interesting drug candidates that mitigate key pathological hallmarks of the disease. This work reviews the most relevant evidence that correlate mitophagy defects and ALS, and discusses the possibility of considering mitophagy as an interesting target in the search for an effective treatment for ALS.
肌萎缩侧索硬化症（ALS）是一种致命的神经退行性疾病，通常在 2 至 4 年内导致呼吸肌麻痹。ALS 的发病机制具有多因素性，病因不明，目前尚无有效治疗方法。绝大多数患者的运动神经元中存在蛋白聚集和功能失调的线粒体堆积。因此，自噬和线粒体自噬调节剂可能是有前途的药物候选物，可以减轻疾病的关键病理特征。本文综述了与线粒体自噬缺陷和 ALS 相关的最相关证据，并讨论了将线粒体自噬作为寻找 ALS 有效治疗方法的一个有意义的靶点的可能性。
