期刊
MOLECULAR GENETICS AND METABOLISM
卷 131, 期 1-2, 页码 1-13出版社
ACADEMIC PRESS INC ELSEVIER SCIENCE
DOI: 10.1016/j.ymgme.2020.10.002
关键词
Mitochondria; Randomized clinical trials; Mitochondrial augmentation; Antioxidants; Mitochondrial biogenesis
资金
- [NIH-5U54-NS078059-09]
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.
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