Rewriting drug-refractory CFTR mutations by prime editing KU Leuven
Specific objectives
1. To develop a prime editing (PE) strategy for the drug-refractory CFTR mutation N1303K
a. To identify the optimal PE system which balances efficiency, with safety and ease of delivery
b. To investigate cell-specific determinants of PE outcomes to establish the most translational model system to measure PE efficiency and outcome purity
2. To engineer Virus Like Particles (VLPs) for transient delivery ...