Tunable Expression of an AAV Payload Using ADAR-mediated RNA Editing
Joseph Silverberg, Laura Pereira, Robert Schmidt, Cameron Baptista, Ahil N. Ganesh, Nicole Harbaugh, Lauren Moffa, A. Metz, Victor Howard, Sean Armour, Daniel M. Cohen, Federico Mingozzi
This paper demonstrates an ADAR-mediated gene switch to control AAV-delivered gene expression by rescuing a premature termination codon via RNA editing, achieving tunable and clinically relevant therapeutic protein levels.
bioRxiv · Aug 5, 2026 · PreprintSynthetic Biology