Functional rescue of F508del-CFTR using small molecule correctors

• Hauptverfasser: Steven eMolinski (Verfasst von), Paul DW Eckford (Verfasst von), Stan ePasyk (Verfasst von), Saumel eAhmadi (Verfasst von), Stephanie eChin (Verfasst von), Christine E. Bear (Verfasst von)
• Format: Buch
• Veröffentlicht: Frontiers Media S.A., 2012-09-01T00:00:00Z.
• Schlagworte: article
• Online-Zugang: 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...