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Welsh, González, and Negulescu share Lasker Award recognizing transformative treatments for people with cystic fibrosis
Benjamin D. Singer, G.R. Scott Budinger
Benjamin D. Singer, G.R. Scott Budinger
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Welsh, González, and Negulescu share Lasker Award recognizing transformative treatments for people with cystic fibrosis

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Abstract

Authors

Benjamin D. Singer, G.R. Scott Budinger

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Figure 1

Mechanisms of action of CFTR modulators.

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Mechanisms of action of CFTR modulators.
(A) Normal CFTR function permit...
(A) Normal CFTR function permits conductance of chloride anions. (B) Class II mutations (e.g., F508del) cause misfolded CFTR and impair trafficking to the apical cell membrane. Class III mutations (e.g., G551D) impair opening (gating) of the CFTR chloride channel. (C) CFTR correctors (e.g., elexacaftor, tezacaftor) promote proper protein folding and trafficking. CFTR potentiators (e.g., ivacaftor) enhance channel opening to promote proper channel gating.

Copyright © 2025 American Society for Clinical Investigation
ISSN: 0021-9738 (print), 1558-8238 (online)

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