Section of Medical Genetics, Children's Hospital, King Fahad Medical City, Riyadh, Saudi Arabia.
Department of Clinical Sciences, College of Medicine, University of Sharjah, Sharjah, United Arab Emirates.
Mol Genet Metab. 2020 Sep-Oct;131(1-2):1-13. doi: 10.1016/j.ymgme.2020.10.002. Epub 2020 Oct 6.
Mitochondrial disorders comprise a molecular and clinically diverse group of diseases that are associated with mitochondrial dysfunction leading to multi-organ disease. With recent advances in molecular technologies, the understanding of the pathomechanisms of a growing list of mitochondrial disorders has been greatly expanded. However, the therapeutic approaches for mitochondrial disorders have lagged behind with treatment options limited mainly to symptom specific therapies and supportive measures. There is an increasing number of clinical trials in mitochondrial disorders aiming for more specific and effective therapies. This review will cover different treatment modalities currently used in mitochondrial disorders, focusing on recent and ongoing clinical trials.
线粒体疾病包括一组分子和临床表现多样的疾病，这些疾病与线粒体功能障碍有关，导致多器官疾病。随着分子技术的最新进展，对越来越多的线粒体疾病的病理机制的理解有了很大的扩展。然而，线粒体疾病的治疗方法却落后了，治疗方法主要局限于针对特定症状的治疗和支持性措施。目前有越来越多的线粒体疾病临床试验旨在寻求更具体和有效的治疗方法。本文将综述目前用于线粒体疾病的不同治疗方式，重点介绍最近和正在进行的临床试验。
