Recent Strategic Advances in CFTR Drug Discovery: An Overview.
International Journal Of Molecular Sciences
Rusnati, Marco M; D'Ursi, Pasqualina P; Pedemonte, Nicoletta N; Urbinati, Chiara C; Ford, Robert C RC; Cichero, Elena E; Uggeri, Matteo M; Orro, Alessandro A; Fossa, Paola P
Requirements for efficient correction of ΔF508 CFTR revealed by analyses of evolved sequences.
Cell
Mendoza, Juan L JL; Schmidt, André A; Li, Qin Q; Nuvaga, Emmanuel E; Barrett, Tyler T; Bridges, Robert J RJ; Feranchak, Andrew P AP; Brautigam, Chad A CA; Thomas, Philip J PJ
Correction of both NBD1 energetics and domain interface is required to restore ΔF508 CFTR folding and function.
Cell
Rabeh, Wael M WM; Bossard, Florian F; Xu, Haijin H; Okiyoneda, Tsukasa T; Bagdany, Miklos M; Mulvihill, Cory M CM; Du, Kai K; di Bernardo, Salvatore S; Liu, Yuhong Y; Konermann, Lars L; Roldan, Ariel A; Lukacs, Gergely L GL
The cystic fibrosis-causing mutation deltaF508 affects multiple steps in cystic fibrosis transmembrane conductance regulator biogenesis.
The Journal Of Biological Chemistry
Thibodeau, Patrick H PH; Richardson, John M JM; Wang, Wei W; Millen, Linda L; Watson, Jarod J; Mendoza, Juan L JL; Du, Kai K; Fischman, Sharon S; Senderowitz, Hanoch H; Lukacs, Gergely L GL; Kirk, Kevin K; Thomas, Philip J PJ