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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Veröffentlicht in:JCI Insight
Hauptverfasser: 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.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society for Clinical Investigation 2018
Schlagworte:
Research Article
Online Zugang: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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