The current landscape for the treatment of mitochondrial disorders

作     者：Jesse Slone Baoheng Gui Taosheng Huang 

作者机构：Division of Human Genetics Cincinnati Children's Hospital Medical Center 3333 Burnet Avenue Cincinnati OH 45229 USA Maternal and Child Health Hospital of Guangxi Zhuang Autonomous Region Nanning 530002 China Human Aging Research Institute Nanchang University Nanchang 330031 China 

出 版 物：《Journal of Genetics and Genomics》 (遗传学报（英文版）)

年 卷 期：2018年第45卷第2期

页      面：71-77页

核心收录：

学科分类：1002[医学-临床医学] 100201[医学-内科学(含：心血管病、血液病、呼吸系病、消化系病、内分泌与代谢病、肾病、风湿病、传染病)] 10[医学] 

主　　题：Mitochondrial disease Genetic disorders Therapy 

摘      要：The mitochondrial organelle is crucial to the energy metabolism of the eukaryotic cell. Defects in mitochondrial function lie at the core of a wide range of disorders, including both rare primary mitochondrial disorders and more common conditions such as Parkinson＇s disease and diabetes. Inherited defects in mitochondrial function can be found in both the nuclear genome and the mitochondrial genome, with the latter creating unique challenges in the treatment and understanding of disease passed on through the mitochondrial genome. In this review, we will describe the limited treatment regimens currently used to alleviate primary mitochondrial disorders, as well as the potential for emerging technologies（in particular, those involving direct manipulation of the mitochondrial genome） to more decisively treat this class of disease. We will also emphasize the critical parallels between primary mitochondrial disorders and more common ailments such as cancer and diabetes.