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TRANSFORMATIVE THERAPIES FOR RARE CFTR MISSENSE ALLELES
With over 1,900 variants reported in the cystic fibrosis transmembrane conductance regulator (CFTR), enhanced understanding of cystic fibrosis (CF) genotype-phenotype correlation represents an important and expanding area of research. The potentiator Ivacaftor has proven an effective treatment for a...
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| Publié dans: | Curr Opin Pharmacol |
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| Auteurs principaux: | , , , |
| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
2017
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| Sujets: | |
| Accès en ligne: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5723219/ https://ncbi.nlm.nih.gov/pubmed/29032041 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.coph.2017.09.018 |
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