Cystic Fibrosis Foundation Therapeutics Announces $7.5 Million Award to Discover New Therapies for Nonsense Mutations

Southern Research will use a sophisticated screening process to identify compounds in its chemical library that could promote the production of functional CFTR protein. In individuals with CF, a defective CFTR protein leads to the buildup of thick mucus, which in turn leads to persistent lung infections and complications in other parts of the body. The University of Alabama at Birmingham (UAB) is collaborating with Southern Research on this five-year project.

CF is a life-threatening, genetic disease that affects approximately 30,000 people in the U.S. and nearly 70,000 people worldwide. About 10 percent of individuals with CF have nonsense mutations. Cystic Fibrosis Foundation Therapeutics has ramped up its efforts to pursue cutting-edge strategies that target these and other mutations by addressing earlier cellular processes that generate the malfunctioning CFTR protein.

Research has shown that nonsense mutations in the CF gene cause messenger RNA molecules to transmit a signal to stop production of the CFTR protein too early. This premature stop signal results in a shortened, and often nonfunctional, CFTR protein.

Using special tests developed by the University of Alabama, Southern Research will look for compounds that would override that premature stop signal, allowing fully functional protein to be made. Southern Research also will look for compounds that would address other problematic cellular processes.

Initial funding through the Alabama Drug Discovery Alliance provided the earlier groundwork for this drug discovery project, which originated with UAB researchers David Bedwell, Ph.D., and Steven M. Rowe, M.D., working with Bob Bostwick, Ph.D., at Southern Research.

Southern Research is a nonprofit organization that focuses on drug discovery and development, engineering, and energy and environment.

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