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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Detalles Bibliográficos
Autores principales: Argyriou, Catherine, Polosa, Anna, Song, Ji Yun, Omri, Samy, Steele, Bradford, Cécyre, Bruno, McDougald, Devin S., Di Pietro, Erminia, Bouchard, Jean-François, Bennett, Jean, Hacia, Joseph G., Lachapelle, Pierre, Braverman, Nancy E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8516995/
https://www.ncbi.nlm.nih.gov/pubmed/34703844
http://dx.doi.org/10.1016/j.omtm.2021.09.002

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