Lipid nanoparticle delivery of the CRISPR/Cas9 system directly into the mitochondria of cells carrying m.7778G>T mutation in MtDNA (mt-Atp8)

Lipid nanoparticle delivery of the CRISPR/Cas9 system directly into the mitochondria of cells carrying m.7778G>T mutation in MtDNA (mt-Atp8)

Abstract Mitochondrial genome mutations are associated with various diseases and gene therapy targeted to mitochondria has the potential to effectively treat such diseases. Here, we targeted a point mutation in mitochondrial DNA (mtDNA) that can cause mitochondrial diseases via delivery of the clust...

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Bibliographic Details
Main Authors: Kaede Norota, Sen Ishizuka, Misa Hirose, Yusuke Sato, Masatoshi Maeki, Manabu Tokeshi, Saleh M. Ibrahim, Hideyoshi Harashima, Yuma Yamada
Format: Article
Language:English
Published: Nature Portfolio 2025-06-01
Series:Scientific Reports
Subjects:
Mitochondrial genome editing
CRISPR/Cas9 ribonucleoprotein (RNP)
Lipid nanoparticle (LNP)
Mitochondrial-targeted delivery
MITO-Porter
Online Access:https://doi.org/10.1038/s41598-025-03671-8
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https://doi.org/10.1038/s41598-025-03671-8

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