Ocular Safety and Efficacy of AAV-mediated Tyrosinase Gene Augmentation in a Nonhuman Primate Model
Jaegook Lim, Alessandra M. Larimer-Picciani, Ala Moshiri, Jui-Kai Wang, Noriyoshi Takahashi, Ana Claudia Santos Raposo, Monica J. Motta, Leah C. Byrne, Sara M. Thomasy
This study evaluates the safety and efficacy of AAV-mediated TYR gene augmentation in nonhuman primates for OCA1 treatment, showing localized hypermelanosis and RPE transduction without severe adverse effects.
bioRxiv · Jul 21, 2026 · PreprintBioengineering