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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Dades bibliogràfiques
Publicat a:JCI Insight
Autors principals: 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
Idioma:Inglês
Publicat: American Society for Clinical Investigation 2018
Matèries:
Research Article
Accés en línia: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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