Clinically translatable mitochondrial gene therapy in muscle using tandem mtZFN architecture

Clinically translatable mitochondrial gene therapy in muscle using tandem mtZFN architecture

Abstract Mutations in the mitochondrial genome (mtDNA) often lead to clinical pathologies. Mitochondrially-targeted zinc finger nucleases (mtZFNs) have been successful in reducing the levels of mutation-bearing mtDNA both in vivo and in vitro, resulting in a shift in the genetic makeup of affected m...

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Main Authors: Pavel A Nash, Keira M Turner, Christopher A Powell, Lindsey Van Haute, Pedro Silva-Pinheiro, Felix Bubeck, Ellen Wiedtke, Eloïse Marques, Dylan G Ryan, Dirk Grimm, Payam A Gammage, Michal Minczuk
Format: Article
Language:English
Published: Springer Nature 2025-04-01
Series:EMBO Molecular Medicine
Subjects:
Gene Therapy
Zinc Finger Nuclease (mtZFN)
Adeno-Associated Viruses (AAV)
mtDNA Heteroplasmy Modification
Skeletal Muscle
Online Access:https://doi.org/10.1038/s44321-025-00231-5
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https://doi.org/10.1038/s44321-025-00231-5

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