《生命科学》 2012, 24(2): 185-190

线粒体相关的疾病治疗及干预策略

郑斌娇¹，梁　敏¹，薛　凌¹，郑　静¹，张　婷¹，龚莎莎¹，方　芳¹，吕建新¹，管敏鑫^1，2*

(1 温州医学院，Attardi线粒体生物医学研究院和浙江省医学遗传学重点实验室，温州 325035；2 浙江大学生命科学学院，杭州 310058)

摘　要：摘　要：线粒体疾病是一种累及不同的组织和器官的复杂异质性疾病，由核基因或线粒体基因的遗传缺陷导致，同时也受环境因素的影响。近十年来，有关线粒体疾病的诊断及发生机制的研究进展迅速，而疾病的治疗方法却研究较少。着重介绍线粒体疾病的相关治疗方法和干预策略。
关键词：线粒体疾病；基因治疗；氧化磷酸化缺陷；异质性

Therapeutic and tampering approaches for mitochondrial associated diseases

ZHENG Bin-Jiao¹， LIANG Min¹， XUE Ling¹， ZHENG Jing¹， ZHANG Ting¹， GONG Sha-Sha1， FANG Fang¹， Lv Jian-Xin¹， GUAN Min-Xin^1，2*

(1 Attardi Institute of Mitochondrial Biomedicine and Zhejiang Provincial Key Laboratory of Medical Genetics， Wenzhou Medical College， Wenzhou 325035， China; 2 College of Life Sciences， Zhejiang University， Hangzhou 310058， China)

Abstract: Abstract: As heterogeneous disorders， mitochondrial diseases can affect various tissues and organs. They can be caused by genetic defects (nuclear and mitochondrial DNA) or environmental factors. Last decades， substantial progress has been made among the diagnosis and pathogenesis of mitochondrial diseases. However， there have been not many reports about development of therapies approaches for these disorders. In this review article， we will discuss emerging therapeutic and intervention strategies of mitochondrial diseases.
Key words: mitochondrial diseases; gene therapy; oxidative phosphorylation defects; heteroplasmy