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The use of a MITO-Porter to deliver exogenous therapeutic RNA to a mitochondrial disease’s cell with a A1555G mutation in the mitochondrial 12S rRNA gene results in an increase in mitochondrial respiratory activity
Mitochondrion ( IF 4.4 ) Pub Date : 2020-11-01 , DOI: 10.1016/j.mito.2020.09.008
Yuma Yamada , Minako Maruyama , Tomoko Kita , Shin-ichi Usami , Shin-ichiro Kitajiri , Hideyoshi Harashima

We report on validating a mitochondrial gene therapeutic strategy using fibroblasts derived from patients with an A1555G point mutation in mitochondrial DNA coding 12S ribosomal RNA (rRNA (12S)).Wild-type rRNA (12S) as a therapeutic RNA was encapsulated in a mitochondrial targeting liposome, a MITO-Porter (rRNA-MITO-Porter), and an attempt was made to deliver the MITO-Porter to mitochondria of the diseased cells. It was confirmed that the rRNA-MITO-Porter treatment significantly decreased the ratio of the mutant rRNA content. Moreover, it was shown that the mitochondrial respiratory activities of the diseased cells were improved as the result of the mitochondrial transfection of the rRNA-MITO-Porter.

中文翻译:

使用 MITO-Porter 将外源治疗性 RNA 递送至线粒体 12S rRNA 基因 A1555G 突变的线粒体疾病细胞,导致线粒体呼吸活动增加

我们报告了使用来自编码 12S 核糖体 RNA (rRNA (12S)) 的线粒体 DNA A1555G 点突变患者的成纤维细胞验证线粒体基因治疗策略。野生型 rRNA (12S) 作为治疗性 RNA 被封装在线粒体靶向中脂质体,一种 MITO-Porter (rRNA-MITO-Porter),并尝试将 MITO-Porter 输送到患病细胞的线粒体。经证实,rRNA-MITO-Porter 处理显着降低了突变 rRNA 含量的比例。此外,表明由于rRNA-MITO-Porter的线粒体转染,患病细胞的线粒体呼吸活动得到改善。
更新日期:2020-11-01
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