Departamento de Bioquímica, Biología Molecular y Celular, Universidad de Zaragoza, 50013 Zaragoza, Spain; Instituto de Investigación Sanitaria de Aragón (IISA), Universidad de Zaragoza, 50013 Zaragoza, Spain; Centro de Investigaciones Biomédicas en Red de Enfermedades Raras (CIBERER), Universidad de Zaragoza, 50013 Zaragoza, Spain; Fundación ARAID, Universidad de Zaragoza, 50013 Zaragoza, Spain.
Departamento de Bioquímica, Biología Molecular y Celular, Universidad de Zaragoza, 50013 Zaragoza, Spain; Instituto de Investigación Sanitaria de Aragón (IISA), Universidad de Zaragoza, 50013 Zaragoza, Spain; Centro de Investigaciones Biomédicas en Red de Enfermedades Raras (CIBERER), Universidad de Zaragoza, 50013 Zaragoza, Spain.
Drug Discov Today. 2018 Mar;23(3):493-498. doi: 10.1016/j.drudis.2018.01.031. Epub 2018 Jan 11.
Leber hereditary optic neuropathy (LHON) is a rare, inherited mitochondrial disease. No treatment has shown a clear-cut benefit on a clinically meaningful end-point. Primary open-angle glaucoma (POAG) is a frequent, acquired optic neuropathy. Lowering intraocular pressure (IOP) reduces disease progression. However, current methods to decelerate this progression are recognized as being inadequate. Therefore, there is a clear need to look for new therapeutic approaches. The growing evidence indicates that POAG can also be a mitochondrial optic neuropathy (MON). Several risk elements are common for both diseases and all of them decrease mitochondrial (mt)DNA content. Based on these susceptibility factors and their molecular mechanism, we suggest herein pharmacological therapies targeted to increase mtDNA levels, oxidative phosphorylation (OXPHOS) capability, and mitochondrial energy production as treatments for MONs.
Leber 遗传性视神经病变（LHON）是一种罕见的遗传性线粒体疾病。目前尚无任何治疗方法能在临床上明确改善终点结局。原发性开角型青光眼（POAG）是一种常见的获得性视神经病变。降低眼内压（IOP）可减缓疾病进展。然而，目前减缓这一进展的方法效果有限。因此，显然需要寻找新的治疗方法。越来越多的证据表明，POAG 也可能是一种线粒体视神经病变（MON）。这两种疾病有几个共同的风险因素，所有这些因素都会导致线粒体（mt）DNA 含量减少。基于这些易感因素及其分子机制，我们建议采用靶向增加 mtDNA 水平、氧化磷酸化（OXPHOS）能力和线粒体能量产生的药物治疗方法，以治疗 MON。
