| RRC ID |
58030
|
| 著者 |
Kawamura E, Hibino M, Harashima H, Yamada Y.
|
| タイトル |
Targeted mitochondrial delivery of antisense RNA-containing nanoparticles by a MITO-Porter for safe and efficient mitochondrial gene silencing.
|
| ジャーナル |
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.
|
| 巻・号 |
49
|
| ページ |
178-188
|
| 公開日 |
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 |
3.992
|
| 引用数 |
1
|
| リソース情報 |
| ヒト・動物細胞 |
HeLa |
