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• W3092533936 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." @default.
• W3092533936 created "2020-10-15" @default.
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• W3092533936 date "2020-11-01" @default.
• W3092533936 modified "2023-10-16" @default.
• W3092533936 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" @default.
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• W3092533936 doi "https://doi.org/10.1016/j.mito.2020.09.008" @default.
• W3092533936 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/33035688" @default.
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