RRC ID 58030
Author Kawamura E, Hibino M, Harashima H, Yamada Y.
Title Targeted mitochondrial delivery of antisense RNA-containing nanoparticles by a MITO-Porter for safe and efficient mitochondrial gene silencing.
Journal Mitochondrion
Abstract Mitochondrial gene therapy will be needed to treat mitochondrial diseases. We previously demonstrated mitochondrial gene silencing by the mitochondrial delivery of antisense RNA oligonucleotide (ASO) targeting mtDNA-encoded mRNA using a MITO-Porter, a liposomal nano carrier system designed for mitochondrial delivery. Here, we report on the efficient packaging of ASO in the MITO-Porter via a nanoparticle packaging method, which showed a 10-fold higher packaging efficiency than the conventional method. The constructed carrier showed a decrease in the target mRNA levels and ATP production. These results indicate that such a MITO-Porter has potential for use in therapies designed to regulate mitochondrial function.
Volume 49
Pages 178-188
Published 2019-11-1
DOI 10.1016/j.mito.2019.08.004
PII S1567-7249(18)30286-1
PMID 31472283
MeSH Gene Silencing* Gene Transfer Techniques* Genes, Mitochondrial* HeLa Cells Humans Mitochondria* / genetics Mitochondria* / metabolism Nanoparticles / chemistry* RNA, Antisense* / chemistry RNA, Antisense* / genetics RNA, Antisense* / pharmacology
IF 3.449
Times Cited 1
Human and Animal Cells HeLa