Small molecule correction for cystic fibrosis

Main Category: Cystic Fibrosis
Article Date: 27 Aug 2005 - 3:00 PDT

email to a friend   printer friendly   opinions  

Cystic fibrosis is one of the most common genetically transmitted diseases. In a study appearing online on August 25 in advance of print publication of the September 1 issue of the Journal of Clinical Investigation, Alan Verkman and colleagues from UCSF report the discovery and characterization of small molecule 'correctors' of defective cystic fibrosis conductance regulator (CFTR) cellular processing. A deletion in phenylalanine 508 is a mutation that disrupts the function of this cellular channel and causes cystic fibrosis.

The authors screen 150,000 compounds using a cell-based functional assay, and test structural analogs of active compounds. Compounds were identified that effectively corrected the mutant CFTR cellular processing biochemically and functionally, and conferred proper channel function to human bronchial cells from cystic fibrosis patients. This work represents a strategy to treat cystic fibrosis, by treating the underlying CFTR defect.

TITLE:Small molecule correctors of defective deltaF508-CFTR cellular processing identified by high-throughput screening

AUTHOR:
Alan Verkman
University of California at San Francisco, San Francisco, CA USA

View the PDF of this article at:
the-jci.org/article.php?id=24898

Stacie Bloom
press_releases@the-jci.org
212-342-4159
Journal of Clinical Investigation
http://www.jci.org

• Additional
• References
• Citations