2025-11-07
Toward an all-in-one recombinant adeno-associated virus vector for functionally ablating the prion gene using CRISPR-Cas technology
2025-11-07 • Claire Verkuyl, Ari Belotserkovsky, Thomas Zerbes, Declan Williams, M. Krishnan, Sabrina Zhu, Sophie Grunnesjӧ, Shehab Eid, Cunjie Zhang, Wenda Zha...
Any strategy that can selectively and persistently lower the brain levels of the cellular prion protein (PrP C ) is expected to extend survival in prion diseases. Recent advances in the virus-mediated delivery of gene therapies prompted us to explore if a recombinant adeno-associated virus (rAAV) vector delivering a CRISPR-Cas-based gene editor can be devised that induces a functional knockout of the prion gene. Whereas the eventual objective is to assess the therapeutic potency of an optimized vector in prion-inf…