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Toward clinical translation of retinal gene therapy for Zellweger Spectrum Disorder

Toward clinical translation of retinal gene therapy for Zellweger Spectrum Disorder
视网膜基因疗法治疗齐薇格谱系障碍的临床转化
批准号：
491823
负责人：
Braverman Nancy E
金额：
$0.0万
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：
2023
资助国家：
加拿大
项目状态：
已结题
起止时间：
2023-09-01 至 2024-09-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=491823&lang=en
关键词：
Aav Manufacturing Cta Enabling Studies Mouse Safety Studies Peroxisome Disorder Retinal Gene Therapy

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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 reduced functions of peroxisomes, organelles found in all body cells. Progressive retinal degeneration leading to blindness is a major untreatable disability,
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