Enabling commercialization of retinal gene therapy for Zellweger Spectrum Disorder

实现齐薇格谱系障碍视网膜基因疗法的商业化

• 批准号: 469606
• 负责人: Braverman Nancy E
• 金额: $ 109.51万
• 依托单位: Research Institute of the McGill University Health Centre
• 依托单位国家: 加拿大
• 项目类别: Operating Grants
• 财政年份: 2022
• 资助国家: 加拿大
• 起止时间: 2022-09-01 至 2027-09-01
• 项目状态: 未结题
• 来源: https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=469606&lang=en
• 关键词: Clinlcal Outcome Measures; Cta Enabling Studies; Mouse Bodistribution; Mouse Toxicity; Peroxisome Disorder; Prospective Natural History Study; Retinal Gene Therapy

Zellweger Spectrum Disorder (ZSD) is commonly caused by mutations in the PEX1 gene, resulting in reduced functions of peroxisomes, organelles found in all body cells. Progressive retinal degeneration leading to blindness is a major untreatable disability,

齐薇格谱系障碍（ZSD）通常由PEX1基因突变引起，导致过氧化物酶体（所有体细胞中都存在的细胞器）功能降低。进行性视网膜变性导致失明是一种无法治愈的主要残疾，