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Developing retinal gene augmentation therapy for Zellweger Spectrum Disorder

Developing retinal gene augmentation therapy for Zellweger Spectrum Disorder
开发针对齐薇格谱系障碍的视网膜基因增强疗法
批准号：
409534
负责人：
Braverman Nancy E
金额：
$67.99万
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：
2019
资助国家：
加拿大
项目状态：
已结题
起止时间：
2019-09-01 至 2024-09-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=409534&lang=en
关键词：
Mouse Model Of Genetic Disease Peroxisome Dysfunction Pex1 Retinal Degeneration Retinal Gene Augmentation Therapy Zellweger Spectrum Disorder

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中文摘要
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英文摘要
Zellweger Spectrum Disorder (ZSD) is commonly caused by mutations in the PEX1 gene, resulting in peroxisome dysfunction. Visual impairment is a major untreatable handicap for ZSD patients, and alleviating this burden would enable improved communication, l
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Toward clinical translation of retinal gene therapy for Zellweger Spectrum Disorder
Enabling commercialization of retinal gene therapy for Zellweger Spectrum Disorder
Getting Ready for Vision Therapy in Peroxisome Biogenesis Disorders
Translating Peroxisome Biogenesis Disorders: Identifying Pharmacological Therapies and Clinical Trial Endpoints (PERescue)
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