Mitochondrial targeted meganuclease as a platform to eliminate mutant mtDNA in vivo
oleh: Ugne Zekonyte, Sandra R. Bacman, Jeff Smith, Wendy Shoop, Claudia V. Pereira, Ginger Tomberlin, James Stewart, Derek Jantz, Carlos T. Moraes
| Format: | Article |
|---|---|
| Diterbitkan: | Nature Portfolio 2021-05-01 |
Deskripsi
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.