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Experimental Strategies Towards Treating Mitochondrial DNA Disorders

机译:治疗线粒体DNA疾病的实验策略

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摘要

extensive range of molecular defects have been identified in the human mitochondrial genome (mtDNA), causing a range of clinical phenotypes characterized by mitochondrial respiratory chain dysfunction. Sadly, given the complexities of mitochondrial genetics, there are no available cures for mtDNA disorders. In this review, we consider experimental, genetic-based strategies that have been or are being explored towards developing treatments, focussing on two specific areas which we are actively pursuing—assessing the benefit of exercise training for patients with mtDNA defects, and the prevention of mtDNA disease transmission.
机译:在人类线粒体基因组(mtDNA)中已经发现了广泛的分子缺陷,导致了一系列以线粒体呼吸链功能异常为特征的临床表型。令人遗憾的是,考虑到线粒体遗传学的复杂性,目前尚无治疗mtDNA疾病的方法。在本综述中,我们考虑已经或正在探索的基于遗传的实验性策略,以开发治疗方法,重点关注我们正在积极追求的两个特定领域-评估mtDNA缺陷患者进行运动训练的益处,以及预防mtDNA疾病传播。

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