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

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

Here, we report on validating a mitochondrial gene therapy by delivering nucleic acids to mitochondria of diseased cells by a MITO-Porter, a liposome-based carrier for mitochondrial delivery. We used cells derived from a patient with a mitochondrial disease with a G625A heteroplasmic mutation in the...

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Bibliographic Details
Main Authors: Eriko Kawamura, Minako Maruyama, Jiro Abe, Akira Sudo, Atsuhito Takeda, Shingo Takada, Takashi Yokota, Shintaro Kinugawa, Hideyoshi Harashima, Yuma Yamada
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
mitochondrial delivery
MITO-Porter
nucleic acids medicine
mitochondrial gene therapy
heteroplasmic mutation
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253120301128

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http://www.sciencedirect.com/science/article/pii/S2162253120301128

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