Prion Diseases

Prion diseases are a related group of rare, fatal brain diseases that affect animals and humans. Also known as transmissible spongiform encephalopathies (TSE), they include bovine spongiform encephalopathy ("mad cow" disease) in cattle; Creutzfeldt-Jakob disease in humans; scrapie in sheep; and chronic wasting disease in deer and elk.

Why is the study of Prion Diseases a priority for NIAID?: 

Much about TSE diseases remains unknown. The diseases are characterized by certain misshapen protein molecules that appear in brain tissue. Normal forms of these prion protein molecules reside on the surface of many types of cells, including brain cells, but scientists do not understand what normal prion protein does. On the other hand, scientists believe that abnormal prion protein, which clumps together and accumulates in brain tissue, is the likely cause of the brain damage that occurs in TSE diseases. Scientists do not have a good understanding of what causes the normal prion protein to take on the misshapen abnormal form.

How is NIAID addressing this critical topic?: 

NIAID conducts TSE disease research in its Rocky Mountain Laboratories in Hamilton, Montana, and also funds prion disease research in university labs. Two other Institutes at the National Institutes of Health also fund prion disease research—the National Institute of Neurological Disorders and Stroke and the National Institute on Aging.

To learn about risk factors for prion diseases and current prevention and treatment strategies visit the Centers for Diseases Control and Prevention (CDC) prion diseases site.

Prion Protein Fibrils

Prion protein expressed in E. coli, purified and fibrillized at pH 7. Recombinant proteins such as the prion protein shown here are often used to model how proteins misfold and sometimes polymerize in neurodegenerative disorders.

Credit: 

NIAID
Biology & Genetics

Scientists are examining how abnormal prion protein molecules cause transmissible spongiform encephalopathy (TSE) diseases. NIAID scientists co-discovered and were among the first to clone the prion protein gene, and also discovered that abnormal prion protein can convert normal prion protein to the abnormal form.

Therapeutic Approaches

Although there are no known ways to cure transmissible spongiform encephalopathy (TSE) diseases, scientists around the world are working to develop treatments. Using infected tissue culture cells for fast initial screening, NIAID researchers have tested thousands of compounds and identified hundreds of molecules that inhibit the formation of the abnormal form of prion protein. Further testing of the most potent of these inhibitors has revealed several that can prolong the lives of rodents if treatments begin soon after infection.

Disease-Specific Research

Prion diseases are a related group of rare, fatal brain diseases that affect animals and humans. Also known as transmissible spongiform encephalopathies (TSE), they include bovine spongiform encephalopathy ("mad cow" disease) in cattle; Creutzfeldt-Jakob disease in humans; scrapie in sheep; and chronic wasting disease in deer and elk.

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