Back to Search
Start Over
Targeted mitochondrial delivery of antisense RNA-containing nanoparticles by a MITO-Porter for safe and efficient mitochondrial gene silencing.
- Source :
-
Mitochondrion . Nov2019, Vol. 49, p178-188. 11p. - Publication Year :
- 2019
-
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. • Antisense RNA was efficiently packaged in the MITO-Porter for mitochondrial delivery. • The constructed carrier showed a decrease in the target mRNA levels. • MITO-Porter could be used in therapies designed to regulate mitochondrial function. [ABSTRACT FROM AUTHOR]
Details
- Language :
- English
- ISSN :
- 15677249
- Volume :
- 49
- Database :
- Academic Search Index
- Journal :
- Mitochondrion
- Publication Type :
- Academic Journal
- Accession number :
- 139904953
- Full Text :
- https://doi.org/10.1016/j.mito.2019.08.004