This program is working to identify potential therapies for people with CF who have nonsense mutations. Nonsense mutations (also known as “x” or “stop” mutations) in the CFTR gene cause the production of CFTR protein to stop prematurely. Overriding these premature stop signals would allow full-length, functional CFTR protein to be made.
Laboratory studies to develop and test these compounds are underway.
This program is sponsored by Icagen, Inc. and partially funded by the Cystic Fibrosis Foundation.
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