Pepe Salvatore, Mentzer Robert M, Gottlieb Roberta A
Heart Research, Murdoch Children's Research Institute, The Royal Children's Hospital, Melbourne, Australia.
J Bioenerg Biomembr. 2014 Aug;46(4):337-45. doi: 10.1007/s10863-014-9559-7. Epub 2014 Jul 9.
Complex I deficiency is difficult to treat because of the size and complexity of the multi-subunit enzyme complex. Mutations or deletions in the mitochondrial genome are not amenable to gene therapy. However, animal studies have shown that yeast-derived internal NADH quinone oxidoreductase (Ndi1) can be delivered as a cell-permeable recombinant protein (Tat-Ndi1) that can functionally replace complex I damaged by ischemia/reperfusion. Current and future treatment of disorders affecting complex I are discussed, including the use of Tat-Ndi1.
由于多亚基酶复合物的规模和复杂性，复合体I缺乏症难以治疗。线粒体基因组中的突变或缺失不适合基因治疗。然而，动物研究表明，酵母来源的内部NADH醌氧化还原酶（Ndi1）可以作为一种细胞可渗透的重组蛋白（Tat-Ndi1）传递，它可以在功能上替代因缺血/再灌注而受损的复合体I。本文讨论了影响复合体I的疾病的当前和未来治疗方法，包括Tat-Ndi1的使用。
