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Enabling commercialization of retinal gene therapy for Zellweger Spectrum Disorder

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
项目状态：
未结题
起止时间：
2022-09-01 至 2027-09-01
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

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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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