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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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Bibliografiske detaljer
Udgivet i:Curr Opin Pharmacol
Main Authors: Oliver, Kathryn E., Han, Sangwoo T., Sorscher, Eric J., Cutting, Garry R.
Format: Artigo
Sprog:Inglês
Udgivet: 2017
Fag:
Online adgang: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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