| RRC ID |
63100
|
| 著者 |
Yamada Y, Maruyama M, Kita T, Usami SI, Kitajiri SI, Harashima H.
|
| タイトル |
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.
|
| ジャーナル |
Mitochondrion
|
| Abstract |
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.
|
| 巻・号 |
55
|
| ページ |
134-144
|
| 公開日 |
2020-11-1
|
| DOI |
10.1016/j.mito.2020.09.008
|
| PII |
S1567-7249(20)30196-3
|
| PMID |
33035688
|
| MeSH |
Cell Line
Cell Respiration
Fibroblasts / cytology
Fibroblasts / metabolism
Humans
Liposomes
Mitochondria / physiology*
Mitochondrial Diseases / genetics*
Mitochondrial Diseases / therapy
Mutation*
RNA, Ribosomal / genetics
RNA, Ribosomal / pharmacology*
Transfection
|
| IF |
3.992
|
| リソース情報 |
| ヒト・動物細胞 |
NB1RGB(RCB0222) |
