Journal
MITOCHONDRION
Volume 49, Issue -, Pages 178-188Publisher
ELSEVIER SCI LTD
DOI: 10.1016/j.mito.2019.08.004
Keywords
Mitochondrial RNA knockdown; Mitochondrial drug delivery; MITO-porter; Nucleic acids delivery; Nucleic acids medicine
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Funding
- Ministry of Education, Culture, Sports, Science and Technology of Japanese Government (MEXT) [26282131, 17H02094, 15H12532]
- Japan Society for the Promotion of Science (JSPS) [14J02379]
- Uehara Memorial Foundation
- Akiyama Life Science Foundation
- Grants-in-Aid for Scientific Research [17H02094, 26282131, 14J02379] Funding Source: KAKEN
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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.
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