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…·Catherine Argyriou
This study evaluates a gene therapy for Zellweger spectrum disorder, showing long-term preservation of retinal function and structure in mice.