Functional rescue of F508del-CFTR using small molecule correctors

• Päätekijät: Steven eMolinski (Tekijä), Paul DW Eckford (Tekijä), Stan ePasyk (Tekijä), Saumel eAhmadi (Tekijä), Stephanie eChin (Tekijä), Christine E. Bear (Tekijä)
• Aineistotyyppi: Kirja
• Julkaistu: Frontiers Media S.A., 2012-09-01T00:00:00Z.
• Aiheet: article
• Linkit: 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...