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Article

Efficient elimination of MELAS-associated m.3243G mutant mitochondrial DNA by an engineered mitoARCUS nuclease.

Nature metabolism - 1 Dec 2023

Shoop Wendy K, Lape Janel, Trum Megan, Powell Alea, Sevigny Emma, Mischler Adam, Bacman Sandra R, Fontanesi Flavia, Smith Jeff, Jantz Derek, Gorsuch Cassandra L, Moraes Carlos T

Abstract excerpt

Nuclease-mediated editing of heteroplasmic mitochondrial DNA (mtDNA) seeks to preferentially cleave and eliminate mutant mtDNA, leaving wild-type genomes to repopulate the cell and shift mtDNA heteroplasmy. Various technologies are available, but many suffer from limitations based on size and/or specificity. The use of ARCUS nucleases, derived from naturally occurring I-CreI, avoids these pitfalls due to their...

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Identifiers

PMID: 38036771
DOI: 10.1038/s42255-023-00932-6
PMCID: PMC10730414

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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