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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Shranjeno v:
Bibliografske podrobnosti
izdano v:JCI Insight
Main Authors: 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
Jezik:Inglês
Izdano: American Society for Clinical Investigation 2018
Teme:
Research Article
Online dostop: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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