生物
粒线体疾病
线粒体DNA
遗传学
线粒体
核基因
基因
基因组
生殖系
人类线粒体遗传学
突变
DNAJA3公司
疾病
线粒体融合
生物信息学
计算生物学
病理
医学
作者
Oliver M. Russell,Gráinne Gorman,Robert N. Lightowlers,D.M. Turnbull
出处
期刊:Cell
[Elsevier]
日期:2020-04-01
卷期号:181 (1): 168-188
被引量:233
标识
DOI:10.1016/j.cell.2020.02.051
摘要
Mitochondrial diseases are clinically heterogeneous disorders caused by a wide spectrum of mutations in genes encoded by either the nuclear or the mitochondrial genome. Treatments for mitochondrial diseases are currently focused on symptomatic management rather than improving the biochemical defect caused by a particular mutation. This review focuses on the latest advances in the development of treatments for mitochondrial disease, both small molecules and gene therapies, as well as methods to prevent transmission of mitochondrial disease through the germline.
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