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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Detalles Bibliográficos
Autores principales: Ugne Zekonyte, Sandra R. Bacman, Jeff Smith, Wendy Shoop, Claudia V. Pereira, Ginger Tomberlin, James Stewart, Derek Jantz, Carlos T. Moraes
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2021
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Acceso en línea:https://doaj.org/article/e7f6099041724090957f81336f63c028
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Descripción
Sumario: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.