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Mutation-specific downregulation of CFTR2 variants by gating potentiators.
Hum Mol Genet. 2017 Dec 15;26(24):4873-4885. doi: 10.1093/hmg/ddx367.
Hum Mol Genet. 2017.
PMID: 29040544
Free PMC article.
Several mutants exhibit functional defects, correctable by gating potentiators. Long-term exposure (24 h) to the only available potentiator drug, VX-770, leads to the biochemical and functional downregulation of F508del-CFTR both in immortalized and primary human airway ce …
Several mutants exhibit functional defects, correctable by gating potentiators. Long-term exposure (24 h) to the only available poten …
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