Clinical trials in mitochondrial disorders, an update

被引：48
作者：
Almannai, Mohammed [1 ]
El-Hattab, Ayman W. [2 ]
Ali, May [3 ]
Soler-Alfonso, Claudia [3 ,4 ]
Scaglia, Fernando [3 ,4 ,5 ]
机构：
[1] King Fahad Med City, Sect Med Genet, Childrens Hosp, Riyadh, Saudi Arabia
[2] Univ Sharjah, Coll Med, Dept Clin Sci, Sharjah, U Arab Emirates
[3] Baylor Coll Med, Dept Mol & Human Genet, Mark Wallace Tower,Suite 1560,6701 Fannin St, Houston, TX 77030 USA
[4] Texas Childrens Hosp, Mark Wallace Tower,Suite 1560,6701 Fannin St, Houston, TX 77030 USA
[5] Prince Wales Hosp, Joint BCM CUHK Ctr Med Genet, Shatin, Hong Kong, Peoples R China
来源：
MOLECULAR GENETICS AND METABOLISM | 2020年 / 131卷 / 1-2期
关键词：
Mitochondria; Randomized clinical trials; Mitochondrial augmentation; Antioxidants; Mitochondrial biogenesis; NITRIC-OXIDE PRODUCTION; OPTIMIZED ALLOTOPIC EXPRESSION; STEM-CELL TRANSPLANTATION; ACTIVATED PROTEIN-KINASE; LONG-TERM OUTCOMES; LIVER-TRANSPLANTATION; SKELETAL-MUSCLE; GENE-THERAPY; MOUSE MODEL; SELECTIVE ELIMINATION;
D O I：
10.1016/j.ymgme.2020.10.002
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
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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页码：1 / 13
页数：13
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