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Validation of a mitochondrial RNA therapeutic strategy using fibroblasts from a Leigh syndrome patient with a mutation in the mitochondrial ND3 gene
| Title: | Validation of a mitochondrial RNA therapeutic strategy using fibroblasts from a Leigh syndrome patient with a mutation in the mitochondrial ND3 gene |
| Authors: | Yamada, Yuma Browse this author →KAKEN DB | | Somiya, Kana Browse this author | | Miyauchi, Akihiko Browse this author | | Osaka, Hitoshi Browse this author | | Harashima, Hideyoshi Browse this author →KAKEN DB |
| Issue Date: | 5-May-2020 |
| Publisher: | Nature Publishing Group |
| Journal Title: | Scientific reports |
| Volume: | 10 |
| Start Page: | 7511 |
| Publisher DOI: | 10.1038/s41598-020-64322-8 |
| Abstract: | We report on the validation of a mitochondrial gene therapeutic strategy using fibroblasts from a Leigh syndrome patient by the mitochondrial delivery of therapeutic mRNA. The treatment involves delivering normal ND3 protein-encoding mRNA as a therapeutic RNA to mitochondria of the fibroblasts from a patient with a T10158C mutation in the mtDNA coding the ND3 protein, a component of the mitochondrial respiratory chain complex I. The treatment involved the use of a liposome-based carrier (a MITO-Porter) for delivering therapeutic RNA to mitochondria via membrane fusion. The results confirmed that the mitochondrial transfection of therapeutic RNA by the MITO-Porter system resulted in a decrease in the levels of mutant RNA in mitochondria of diseased cells based on reverse transcription quantitative PCR. An evaluation of mitochondrial respiratory activity by respirometry also showed that transfection using the MITO-Porter resulted in an increase in maximal mitochondrial respiratory activity in the diseased cells. |
| Type: | article |
| URI: | http://hdl.handle.net/2115/78558 |
| Appears in Collections: | 薬学研究院 (Faculty of Pharmaceutical Sciences) > 雑誌発表論文等 (Peer-reviewed Journal Articles, etc)
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