Trikafta restores thermodynamic coupling between two nucleotide binding domains for potentiating CFTR activity.

Abstract excerpt

Trikafta modulators can correct the folding, thermal and gating defects of the most common cystic fibrosis mutant F508del of the human cystic fibrosis transmembrane conductance regulator. While folding correctors VX-445 and VX-809 are sufficient to rescue its folding and thermal defects by restoring Mg/ATP mediated dimerization between the two nucleotide binding domains (NBD1 and NBD2), the thermodynamic basis...

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