Systemic, RPE-directed AAV-tyrosinase therapy restores ocular pigmentation in an OCA1 mouse model
Molecular Therapy Advances 34, 201861 (2026)
Published 7 October 2026Open access
Compares intravenous, subretinal and intravitreal delivery of AAV-tyrosinase with ubiquitous and RPE-directed promoters in an OCA1 mouse model. Early intravenous delivery of the RPE-directed construct restored widespread ocular pigment with lower off-target expression, and treated mice showed altered light-avoidance behavior in a pilot study.
Citation
Larimer-Picciani AM, Jacob LB, Sullinger KJ, Kriebel WG, Sahel J-A, Byrne LC. Systemic, RPE-directed AAV-tyrosinase therapy restores ocular pigmentation in an OCA1 mouse model. Mol Ther Adv. 2026;34:201861. doi:10.1016/j.omta.2026.201861
BibTeX
@article{larimerpicciani2026systemic,
title = {Systemic, RPE-directed AAV-tyrosinase therapy restores ocular pigmentation in an OCA1 mouse model},
author = {Larimer-Picciani, Alessandra M. and Jacob, Lucas B. and Sullinger, Kacey J. and Kriebel, Wyatt G. and Sahel, José-Alain and Byrne, Leah C.},
journal = {Molecular Therapy Advances},
volume = {34},
pages = {201861},
year = {2026},
doi = {10.1016/j.omta.2026.201861}
}