Coordinated regulation using small-molecule drugs enables controlled therapeutic genome editing and enhanced genomic precision in situ - PubMed
2 hours ago
- #precision-medicine
- #therapeutic-applications
- #genome-editing
- The PRINCE system uses small-molecule drugs for controlled therapeutic genome editing with temporal precision.
- It achieves reduced off-target activity compared to constitutive editors across different nuclease types and delivery methods.
- In humanized mouse models, it effectively treated hypercholesterolemia and neovascular age-related macular degeneration in situ.