Future of human mitochondrial DNA editing technologies
Future of human mitochondrial DNA editing technologies
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DOI：
10.1080/24701394.2018.1472773
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发表时间：
2019-02
影响因子：
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通讯作者：
N. Verechshagina;N. Nikitchina;Y. Yamada;Н. Harashima;M. Tanaka;K. Orishchenko;I. Mazunin
中科院分区：
文献类型：
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作者：
N. Verechshagina;N. Nikitchina;Y. Yamada;Н. Harashima;M. Tanaka;K. Orishchenko;I. Mazunin
摘要ATP和其他代谢物是机体细胞发育、维持和功能所必需的，它们都是在线粒体中合成的。存在于线粒体内的基因组的多个拷贝，连同其母系遗传，决定了线粒体DNA（mtDNA）突变的临床表现和传播。深入了解线粒体生物学和开发线粒体疾病基因治疗方法的主要障碍是缺乏允许直接改变mtDNA序列的系统。在这里，我们讨论了操纵mtDNA异质性水平的现有方法，以及未来可用作人类线粒体基因组编辑工具的最新系统。
Abstract ATP and other metabolites, which are necessary for the development, maintenance, and functioning of bodily cells are all synthesized in the mitochondria. Multiple copies of the genome, present within the mitochondria, together with its maternal inheritance, determine the clinical manifestation and spreading of mutations in mitochondrial DNA (mtDNA). The main obstacle in the way of thorough understanding of mitochondrial biology and the development of gene therapy methods for mitochondrial diseases is the absence of systems that allow to directly change mtDNA sequence. Here, we discuss existing methods of manipulating the level of mtDNA heteroplasmy, as well as the latest systems, that could be used in the future as tools for human mitochondrial genome editing.