A CRISPR-based mitochondrial gene therapy tool derived by engineering guide RNAs - PubMed
8 days ago
- #gene therapy
- #mitochondrial DNA
- #CRISPR
- A CRISPR-based mitochondrial gene therapy tool was developed by engineering guide RNAs to target mitochondrial DNA (mtDNA).
- The tool utilizes a 30 nt ST2-RNA mitochondrial targeting sequence (RMTS) derived from lncRNA RP11-46H11.3 for efficient mitochondrial localization.
- RMTS-CRISPR demonstrated heteroplasmic mtDNA shifting efficiencies of up to 26.37% in cell models and 26.79% in vivo.
- This technology offers a potential approach for correcting heterogeneous mtDNA mutations in mitochondrial disorders.