Residual function of cystic fibrosis mutants predicts response to small molecule CFTR modulators

Treatment of individuals with cystic fibrosis (CF) has been transformed by small molecule therapies that target select pathogenic variants in the CF transmembrane conductance regulator (CFTR). To expand treatment eligibility, we stably expressed 43 rare missense CFTR variants associated with moderat...

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Gepubliceerd in:JCI Insight
Hoofdauteurs: Han, Sangwoo T., Rab, Andras, Pellicore, Matthew J., Davis, Emily F., McCague, Allison F., Evans, Taylor A., Joynt, Anya T., Lu, Zhongzhou, Cai, Zhiwei, Raraigh, Karen S., Hong, Jeong S., Sheppard, David N., Sorscher, Eric J., Cutting, Garry R.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: American Society for Clinical Investigation 2018
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Research Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6124440/
https://ncbi.nlm.nih.gov/pubmed/30046002
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.121159
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