线粒体DNA
生物
基因组
线粒体
遗传学
核基因
核DNA
粒线体疾病
计算生物学
基因组工程
线粒体融合
人类线粒体遗传学
基因组编辑
基因
作者
Pedro Silva-Pinheiro,Michal Minczuk
标识
DOI:10.1038/s41576-021-00432-x
摘要
Mitochondria are subject to unique genetic control by both nuclear DNA and their own genome, mitochondrial DNA (mtDNA), of which each mitochondrion contains multiple copies. In humans, mutations in mtDNA can lead to devastating, heritable, multi-system diseases that display different tissue-specific presentation at any stage of life. Despite rapid advances in nuclear genome engineering, for years, mammalian mtDNA has remained resistant to genetic manipulation, hampering our ability to understand the mechanisms that underpin mitochondrial disease. Recent developments in the genetic modification of mammalian mtDNA raise the possibility of using genome editing technologies, such as programmable nucleases and base editors, for the treatment of hereditary mitochondrial disease.
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