Experimental strategies towards treating mitochondrial DNA disorders
Experimental strategies towards treating mitochondrial DNA disorders
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DOI：
10.1007/s10540-007-9042-3
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发表时间：
2007-06-01
影响因子：
4
通讯作者：
Taylor, Robert W.
中科院分区：
文献类型：
--
作者：
Gardner, Julie L.;Craven, Lyndsey;Taylor, Robert W.
在人类线粒体基因组(MtDNA)中发现了广泛的分子缺陷，导致以线粒体呼吸链功能障碍为特征的一系列临床表型。遗憾的是，考虑到线粒体遗传学的复杂性，目前还没有治疗线粒体DNA疾病的有效方法。在这篇综述中，我们考虑已经或正在探索的基于遗传的实验性策略来开发治疗方法，重点放在我们正在积极追求的两个特定领域-评估运动训练对mtDNA缺陷患者的好处，以及防止mtDNA疾病的传播。
An 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.