Article

Clinically relevant AAV8- <i>PEX1</i> gene therapy preserves retinal integrity and function long-term in a murine model of Zellweger spectrum disorder

2026-05-14

Abstract excerpt

Inherited retinal diseases (IRDs) are a heterogeneous group of genetic disorders that cause progressive vision loss. A subset of IRDs is associated with ubiquitously expressed genes involved in fundamental cellular processes, often resulting in multisystem disease. Among these is Zellweger spectrum disorder (ZSD), caused by pathogenic variants in PEX genes required for peroxisome biogenesis and function. There ar...

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Literature Corpus work: b157fe7b-0854-5911-b588-9a1c38d178c8
DOI: 10.64898/2026.05.11.723906

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