NeuroscienceNucleic Acids ResearchPeer-reviewed
Prion protein lowering is a disease-modifying therapy across prion strains, disease stages, and endpoints
Eric Vallabh Minikel, Hien Tran Zhao, Jason Le…·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.
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