AAV-mediated PEX1 gene augmentation improves visual function in the PEX1-Gly844Asp mouse model for mild Zellweger spectrum disorder

AAV-mediated PEX1 gene augmentation improves visual function in the PEX1-Gly844Asp mouse model for mild Zellweger spectrum disorder

Patients with Zellweger spectrum disorder (ZSD) commonly present with vision loss due to mutations in PEX genes required for peroxisome assembly and function. Here, we evaluate PEX1 retinal gene augmentation therapy in a mouse model of mild ZSD bearing the murine equivalent (PEX1-p[Gly844Asp]) of th...

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Bibliographic Details
Main Authors: Catherine Argyriou, Anna Polosa, Ji Yun Song, Samy Omri, Bradford Steele, Bruno Cécyre, Devin S. McDougald, Erminia Di Pietro, Jean-François Bouchard, Jean Bennett, Joseph G. Hacia, Pierre Lachapelle, Nancy E. Braverman
Format: Article
Language:English
Published: Elsevier 2021-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
peroxisome biogenesis disorder
Zellweger spectrum disorder
PEX1
retinal gene therapy
AAV therapy
metabolic disorder gene therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121001376

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http://www.sciencedirect.com/science/article/pii/S2329050121001376

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