Department of Chemical and Biological Engineering, Sookmyung Women's University, Seoul 04310, Korea.
Institute of Advanced Materials and Systems, Sookmyung Women's University, Seoul 04310, Korea.
Molecules. 2018 Sep 11;23(9):2316. doi: 10.3390/molecules23092316.
Mitochondria are the energy-producing organelles of cells. Mitochondrial dysfunctions link to various syndromes and diseases including myoclonic epilepsy and ragged-red fiber disease (MERRF), Leigh syndrome (LS), and Leber hereditary optic neuropathy (LHON). Primary mitochondrial diseases often result from mutations of mitochondrial genomes and nuclear genes that encode the mitochondrial components. However, complete intracellular correction of the mutated genetic parts relevant to mitochondrial structures and functions is technically challenging. Instead, there have been diverse attempts to provide corrected genetic materials with cells. In this review, we discuss recent novel physical, chemical and biological strategies, and methods to introduce genetic cargos into mitochondria of eukaryotic cells. Effective mitochondria-targeting gene delivery systems can reverse multiple mitochondrial disorders by enabling cells to produce functional mitochondrial components.
线粒体是细胞的能量产生细胞器。线粒体功能障碍与多种综合征和疾病有关，包括肌阵挛性癫痫伴破碎红纤维病（MERRF）、 Leigh 综合征（LS）和 Leber 遗传性视神经病变（LHON）。原发性线粒体疾病通常是由线粒体基因组和编码线粒体成分的核基因突变引起的。然而，完全纠正与线粒体结构和功能相关的突变遗传部分在技术上具有挑战性。相反，人们已经尝试了多种方法来为细胞提供纠正后的遗传物质。在这篇综述中，我们讨论了最近用于将遗传物质递送到真核细胞线粒体的新型物理、化学和生物学策略及方法。有效的靶向线粒体的基因传递系统可以通过使细胞产生功能性线粒体成分来逆转多种线粒体疾病。
