Targeted mitochondrial delivery of antisense RNA-containing nanoparticles by a MITO-Porter for safe and efficient mitochondrial gene silencing.
MITO-porter
Mitochondrial RNA knockdown
Mitochondrial drug delivery
Nucleic acids delivery
Nucleic acids medicine
Journal
Mitochondrion
ISSN: 1872-8278
Titre abrégé: Mitochondrion
Pays: Netherlands
ID NLM: 100968751
Informations de publication
Date de publication:
11 2019
11 2019
Historique:
received:
29
11
2018
revised:
09
08
2019
accepted:
19
08
2019
pubmed:
1
9
2019
medline:
7
5
2020
entrez:
1
9
2019
Statut:
ppublish
Résumé
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.
Identifiants
pubmed: 31472283
pii: S1567-7249(18)30286-1
doi: 10.1016/j.mito.2019.08.004
pii:
doi:
Substances chimiques
RNA, Antisense
0
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
178-188Informations de copyright
Copyright © 2019 Elsevier B.V. and Mitochondria Research Society. All rights reserved.