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Article

Gene Therapy Tools for Diseases Caused by Mutations of the Mitochondrial Genome.

International journal of molecular sciences - 18 Jun 2026

Simonov Vladislav, Rastorguev Sergey

Abstract excerpt

Mitochondrial DNA (mtDNA) mutations are associated with a diverse spectrum of diseases and pose a significant threat to human health. Despite their importance as therapeutic targets, the unique structural and electrochemical properties of mitochondria-most notably the impermeable inner mitochondrial membrane and the high membrane potential-present formidable challenges for the targeted delivery of therapeutic...

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DNA, Mitochondrial
Gene Editing
Genetic Therapy
Genome, Mitochondrial
Mitochondria

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Mitochondrial Diseases

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Identifiers

PMID: 42353231
DOI: 10.3390/ijms27125517
PMCID: PMC13299692

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 1 Sept 2026.

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