Prion protein lowering is a disease-modifying therapy across prion strains, disease stages, and endpoints
Eric Vallabh Minikel, Hien Tran Zhao, Jason Le, Jill O’Moore, Rose Pitstick, Samantha Graffam, George A. Carlson, Michael P. Kavanaugh, Jasna Križ, Jae Beom Kim, Jiyan Ma, Holger Wille, Judd M. Aiken, Deborah McKenzie, Katsumi Doh‐ura, , Sonia M. Vallabh
This study shows that lowering prion protein expression with ASOs is effective in treating prion disease across different strains, treatment times, and endpoints.
Nucleic Acids Research · Jul 13, 2020Peer-reviewedNeuroscience