Drug Delivery Research Laboratory, Department of Pharmaceutical Sciences, Dr. H. S. Gour University, Sagar-470003, India.
Curr Gene Ther. 2009 Dec;9(6):475-86. doi: 10.2174/156652309790031102.
Mitochondrial genetics has become an emerging area of research in the field of modern therapeutics. Mitochondrial genome is the source of 13 polypeptides which are components of subunits of complexes of electron transport chain and are used in the generation of ATP by oxidative phosphorylation. Any mutation and/or defects in these mitochondrial genes may cause diseases ranging from neurodegenerative diseases, diabetes mellitus to cancer. In an ideal condition mtDNA should be mutation free. There are various mechanisms for the repair of diseased cell or mitochondrial DNA. Nowadays, nucleic acid based therapeutics has become of interest and represent a new area of research. However, problem consistently encountered is safe and effective delivery of DNA to the mitochondria for therapeutic benefits. There are numerous barriers which are to be surpassed for successful delivery of nucleic acid to the cell interior and ultimately to the mitochondria. For efficient and effective DNA delivery to the mitochondrial matrix, a suitable carrier system is required to be designed and developed. In the present review we have discussed briefly about mitochondrial DNA and related diseases, various barriers encountered in the delivery of DNA, internalization processes, delivery strategies and methods for targeted delivery of DNA to the mitochondria.
线粒体遗传学已成为现代治疗学领域的一个新兴研究领域。线粒体基因组是 13 种多肽的来源，这些多肽是电子传递链复合物亚基的组成部分，用于通过氧化磷酸化产生 ATP。这些线粒体基因的任何突变和/或缺陷都可能导致从神经退行性疾病、糖尿病到癌症等各种疾病。在理想条件下，mtDNA 应该没有突变。有多种机制可以修复病变细胞或线粒体 DNA。如今，基于核酸的治疗已引起关注，代表了一个新的研究领域。然而，始终存在的问题是如何安全有效地将 DNA 递送到线粒体以实现治疗效果。为了将核酸有效地递送到细胞内部并最终递送到线粒体，需要克服许多障碍。为了将 DNA 高效、有效地递送到线粒体基质中，需要设计和开发合适的载体系统。在本综述中，我们简要讨论了线粒体 DNA 及其相关疾病、DNA 递送上遇到的各种障碍、内化过程、递药策略和靶向递送到线粒体的方法。
