Systemic, RPE-directed AAV-Tyrosinase therapy restores ocular pigmentation in an OCA1 mouse model
Alessandra M. Larimer-Picciani, Lucas B Jacob, Kacey J. Sullinger, Wyatt G Kriebel, José‐Alain Sahel, Leah C. Byrne
This study demonstrates that systemic delivery of an RPE-directed AAV-Tyr vector restores ocular pigmentation and reduces photophobia in an OCA1 mouse model.
bioRxiv · Jul 2, 2026 · PreprintMolecular Biology