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Article

Functional Rescue of F508del-CFTR Using Small Molecule Correctors

1 Jan 2012

Abstract excerpt

High-throughput screens for small molecules that are effective in "correcting" the functional expression of F508del-CFTR have yielded several promising hits. Two such compounds are currently in clinical trial. Despite this success, it is clear that further advances will be required in order to restore 50% or greater of wild-type CFTR function to the airways of patients harboring the F508del-CFTR protein. Progress...

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Share to Advanced biosensing and bioanalysis techniques
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PMID: 23055971
DOI: 10.3389/fphar.2012.00160

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