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

doi:10.1016/j.omtn.2020.04.004

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

Faculty of Medical Sciences

Research output: Contribution to journal › Article › peer-review

56 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 language	English
Pages (from-to)	687-698
Number of pages	12
Journal	Molecular Therapy Nucleic Acids
Volume	20
DOIs	https://doi.org/10.1016/j.omtn.2020.04.004
Publication status	Published - Jun 5 2020

All Science Journal Classification (ASJC) codes

Molecular Medicine
Drug Discovery

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10.1016/j.omtn.2020.04.004

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

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.",

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

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AU - Kawamura, Eriko

AU - Maruyama, Minako

AU - Abe, Jiro

AU - Sudo, Akira

AU - Takeda, Atsuhito

AU - Takada, Shingo

AU - Yokota, Takashi

AU - Kinugawa, Shintaro

AU - Harashima, Hideyoshi

AU - Yamada, Yuma

PY - 2020/6/5

Y1 - 2020/6/5

N2 - 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.

AB - 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.

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JO - Molecular Therapy Nucleic Acids

JF - Molecular Therapy Nucleic Acids

ER -

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

Abstract

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