A self-complementary recombinant adeno-associated virus vector coding for an anchorless prion protein carrying the G127V mutation extends survival in a rodent prion disease model
Thomas Zerbes, Claire E. Verkuyl, Cunjie Zhang, Sophie Grunnesjoe, Shehab Eid, Hamza Arshad, Wenda Zhao, Zahra Nasser, Teaghan O’Shea, Ari Bel, Lise Lamoureux, Kathy L. Frost, Jennifer Myskiw, Le yao Li, Erica Stuart, , Gerold Schmitt‐Ulms
This paper demonstrates that delivering a protective prion protein variant via gene therapy extends survival in a prion disease mouse model.
PLOS Pathogens · Aug 4, 2026Peer-reviewedNeuroscience