Beyond Kaftrio: mechanistic insights to maximize N1303K-CFTR rescue
Iwona Maria Pranke, Valeria Capurro, B. Chevalier, Emanuela Pesce, Valeria Tomati, Cristina Pastorino, Aurélie Hatton, Saïk Urien, Mariateresa Lena, Elise Dréano, Renata Bocciardi, Federico Zara, Stefano Pantano, Vito Terlizzi, Cristina Lucanto, , Nicoletta Pedemonte
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