Clinically relevant AAV8-PEX1 gene therapy preserves retinal integrity and function long-term in a murine model of Zellweger spectrum disorder
Samy Omri, Erminia Di Pietro, Devin S. McDougald, Shaghayegh Zokaei, Jean Bennett, Joseph G. Hacia, Nancy Braverman, Catherine Argyriou
This study evaluates a gene therapy for Zellweger spectrum disorder, showing long-term preservation of retinal function and structure in mice.
bioRxiv · May 14, 2026 · PreprintGenetics