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
Abstract
. These results support the conclusion that the mitochondrial delivery of therapeutic nucleic acids represents a viable strategy for mitochondrial gene therapy.
Topics & Concepts
HeteroplasmyMitochondrial DNAMitochondrionGenetic enhancementTransfectionBiologyMutantMitochondrial diseaseNucleic acidMolecular biologyGeneMT-RNR1MutationGene deliveryCell biologyGeneticsMitochondrial Function and PathologyATP Synthase and ATPases ResearchRNA and protein synthesis mechanisms