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
This study demonstrates that a self-complementary recombinant adeno-associated virus vector delivering a GPI-anchorless prion protein with the protective G127V mutation significantly extends survival in a rodent prion disease model by slowing proteomic perturbations, thereby establishing a promising framework for future human gene therapies.