AAV-mediated PEX1 gene augmentation improves visual function in the PEX1-Gly844Asp mouse model for mild Zellweger spectrum disorder
AAV 介导的 PEX1 基因增强可改善轻度 Zellweger 谱系障碍的 PEX1-Gly844Asp 小鼠模型中的视觉功能
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:
doi:10.1016/j.omtm.2021.09.002
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