Experimental Treatment Slows Prion Disease, Extends Life of Mice | NIH: National Institute of Allergy and Infectious Diseases
In these images of mouse brain infected with scrapie, the image on the left was treated with an inactive ASO and shows prion accumulation (brown). The image on the right was treated with an active ASO and shows little accumulation at a comparable timepoint.
Scientists using an experimental treatment have slowed the progression of scrapie, a degenerative central nervous disease caused by prions, in laboratory mice and greatly extended the rodents’ lives, according to a new report in JCI Insight. The scientists used antisense oligonucleotides (ASOs), synthetic compounds that inhibit the formation of specific proteins.
Prion diseases occur when normally harmless prion protein molecules become abnormal and gather in clusters and filaments in the body, including the brain. The diseases are thought to be always fatal. Scrapie, which affects sheep and goats and can be adapted to rodents, is closely related to human prion diseases such as Creutzfeldt-Jakob disease, which is currently untreatable. Thus, scrapie is a valuable experimental model for the development of human prion disease therapies.