Nature Communications (May 2021)

Mitochondrial targeted meganuclease as a platform to eliminate mutant mtDNA in vivo

  • Ugne Zekonyte,
  • Sandra R. Bacman,
  • Jeff Smith,
  • Wendy Shoop,
  • Claudia V. Pereira,
  • Ginger Tomberlin,
  • James Stewart,
  • Derek Jantz,
  • Carlos T. Moraes

DOI
https://doi.org/10.1038/s41467-021-23561-7
Journal volume & issue
Vol. 12, no. 1
pp. 1 – 11

Abstract

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Heteroplasmic mitochondrial DNA mutations lack effective treatments. Here the authors adapt I-CreI meganuclease to target the mitochondria and specifically-eliminate mtDNA with a m.5024C>T mutation in the mttRNA Ala gene.