Redefining hypo- and hyper-responding phenotypes of CFTR mutants for understanding and therapy.
T. Hillenaar, J. Beekman, P. van der Sluijs, I. Braakman
The paper identifies biochemical and functional characteristics of CFTR mutants that respond poorly or exceptionally well to modulators, informing improved therapeutic strategies.
bioRxiv · Sep 12, 2022Biochemistry