Genistein and Analogues for Treatment of Cystic Fibrosis

University of Missouri System: University of Missouri-Columbia
posted on 06/24/2011

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Cystic fibrosis is a devastating disease characterized by progressive disability and is one of the most common fatal genetic disorders among Caucasians in USA and Europe. It is caused by mutations in a channel protein on the cell surface called the cystic fibrosis transmembrane conductance regulator (CFTR). CFTR transports chloride ions across the cell’s plasma membrane. A large number of different mutations in CFTR can cause cystic fibrosis, and the mutations typically either reduce the amount of CFTR on the cell surface, or they reduce the transport activity of the CFTR protein found at the cell surface.

The current invention developed by researchers at the University of Missouri is a method for the treatment of cystic fibrosis by restoring the chloride channel function in cells with mutant CFTR protein using genistein, and derivatives and analogues thereof. Genistein is assumed to function as a potentiator by enhancing the chloride transport of mutant CFTR protein found in the plasma membrane.

Treatment of cystic fibrosis

• A naturally occurring isoflavone, found in soy
  
• Extensively tested for a variety of diseases, including several cancers