Functional rescue of F508del-CFTR using small molecule correctors

• Main Authors: Steven eMolinski (Author), Paul DW Eckford (Author), Stan ePasyk (Author), Saumel eAhmadi (Author), Stephanie eChin (Author), Christine E. Bear (Author)
• Format: Book
• Published: Frontiers Media S.A., 2012-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...