Aug 2026
Leveraging host cell modulators of adeno-associated vectors transduction to tailor viral biodistribution.
A novel approach to detarget liver transduction is developed by transiently downregulating the expression of key entry factors in this tissue using GalNac-siRNAs prior to AAV9 administration, which blunted hepatic transduction but also redirected the vector to other transduction-permissive tissues.
K. Kubek-Luck, J. Velazquez, Xiao-Rui Yao et al.
· Molecular Therapy · 0 citations