| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 8964806 | Journal of Cystic Fibrosis | 2018 | 9 Pages |
Abstract
N1303K has a severe gating defect, reduced ATP-dependence and aberrant response to ATP analogs. These results suggest a defective function of the NBDs in N1303K-CFTR. An improvement of channel function by GLPG1837 or VX-770 and an increase of Band C protein by VX-809 or VX-661 support a therapeutic strategy of combining CFTR potentiator and corrector for patients carrying the N1303K mutation.
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Authors
Samantha DeStefano, Maarten Gees, Tzyh-Chang Hwang,