Molecular BiologybioRxivPreprint
Systemic, RPE-directed AAV-Tyrosinase therapy restores ocular pigmentation in an OCA1 mouse model
Alessandra M. Larimer-Picciani, Lucas B Jacob, Kacey J. Sullinger…·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.
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