Validation of Gene Therapy for Mutant Mitochondria by Delivering Mitochondrial RNA Using a MITO-Porter

Eriko Kawamura, Minako Maruyama, Jiro Abe, Akira Sudo, Atsuhito Takeda, Shingo Takada, Takashi Yokota, Shintaro Kinugawa, Hideyoshi Harashima, Yuma Yamada

Research output: Contribution to journalArticlepeer-review

12 Citations (Scopus)

Abstract

A mitochondrial gene therapy was validated by mitochondrial delivery of a therapeutic RNA in diseased cells with a mutation in the mitochondrial RNA using a MITO-Porter, a nanocarrier for mitochondrial delivery. Mitochondrial transfection of the therapeutic RNA decreased the mutation rate and improved mitochondrial function of the disease cells.

Original languageEnglish
Pages (from-to)687-698
Number of pages12
JournalMolecular Therapy - Nucleic Acids
Volume20
DOIs
Publication statusPublished - Jun 5 2020
Externally publishedYes

All Science Journal Classification (ASJC) codes

  • Molecular Medicine
  • Drug Discovery

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