Mitochondrial targeted meganuclease as a platform to eliminate mutant mtDNA in vivo
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.
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Autores principales: | Ugne Zekonyte, Sandra R. Bacman, Jeff Smith, Wendy Shoop, Claudia V. Pereira, Ginger Tomberlin, James Stewart, Derek Jantz, Carlos T. Moraes |
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Formato: | article |
Lenguaje: | EN |
Publicado: |
Nature Portfolio
2021
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Materias: | |
Acceso en línea: | https://doaj.org/article/e7f6099041724090957f81336f63c028 |
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