Full Rescue of F508del-CFTR Processing and Function by CFTR Modulators can be Achieved by Removal of Two Unique Regulatory Regions
I. Uliyakina, A. C. Da Paula, S. C. Afonso, M. J. Lobo, V. Felicio, H. M. Botelho, C. M. Farinha, M. D. Amaral
This paper investigates how removing regulatory regions in CFTR affects F508del-CFTR rescue by modulators, finding that RI impacts processing while RE affects stability and function.
bioRxiv · May 11, 2018Biochemistry