线粒体基因组靶向编辑研究进展

Progress of targeted gene editing for mitochondrial DNA

线粒体作为细胞中重要的细胞器,其携带有自身的基因组。线粒体基因组发生突变后,可能导致人类疾病。开发靶向线粒体基因组的新型基因编辑工具在疾病治疗和疾病模型构建中具有重要意义。该文总结了线粒体基因编辑领域的进展,特别是近期以DdCBE和TALED为代表的重大进展;同时,也指出目前线粒体基因编辑工具仍然有一些缺点。该文拟为新型线粒体基因编辑工具的研发提供参考。

Mitochondria act as an important organelle in cells,which harbor their own genomes.Mutations in mitochondrial DNA cause human genetic diseases.The development of novel gene-editing tools that target the mitochondrial genome hold great promise for human gene therapy and disease model generation.In the present paper,we summarize the recent progress of mitochondrial gene editing,especially the major technological inventions represented by DdCBE and TALED.At present,there are still some limitations of mitochondrial gene editing tools.The present paper intends to provide more insights for the development of novel gene editing tools to manipulate mitochondrial DNA.