Beyond Kaftrio: mechanistic insights to maximize N1303K-CFTR rescue
I. Pranke, V. Capurro, B. Chevalier, E. Pesce, V. Tomati, C. Pastorino, A. Hatton, S. Urien, M. Lena, E. Dreano, R. Bocciardi, F. Zara, S. Pantano, V. Terlizzi, C. Lucanto, , S. Bui
This study investigates the mechanism of N1303K-CFTR rescue by ELX/TEZ and ETI combotherapy, showing increased protein amounts and activity with potential clinical benefits.
bioRxiv · Feb 29, 2024 · PreprintPhysiology