OHSU researchers develop new drug approach with far-reaching treatment potential

Misfolded protein molecules, caused by gene mutation, are capable of maintaining their function but are misrouted within the cell and can’t work normally, thus causing disease. An OHSU team has discovered a way to use small molecules that enter cells, fix the misfolded proteins and allow the proteins to move to the correct place and function normally again.

The team, led by P. Michael Conn, Ph.D., formerly a senior scientist in reproductive sciences at neurosciences at the Oregon National Primate Research Center, has demonstrated in mice what could be a revolutionary new technique to cure a wide range of human diseases caused by an accumulation of misfolded proteins. Among the potential diseases are neurodegenerative diseases like Alzheimer’s disease, Parkinson’s disease and Huntington’s disease. Other diseases include certain types of diabetes, inherited cataracts and cystic fibrosis.

Dr. Conn recently joined Texas Tech University Health Sciences Center as senior vice president for research and associate provost after 19 years of service at OHSU. His team’s work will be published this week in the early online edition of the Proceedings of the National Academy of Sciences.

View the full OHSU News release.

Research funded by the National Institutes of Health (grants OD012220 and DK85040), the Ben F. Love Endowment, the American Heart Association, the Texas Heart Institute and the Robert A. Welch Foundation

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