RRC ID 63100
Author Yamada Y, Maruyama M, Kita T, Usami SI, Kitajiri SI, Harashima H.
Title 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.
Journal 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.
Volume 55
Pages 134-144
Published 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
Human and Animal Cells NB1RGB(RCB0222)