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Investigating the role of POLR3 in oligodendrocyte-lineage development and myelination in vivo: Translational disease models and proof-of-concept for using in vivo gene therapy to treat POLR3-related Hypomyelinating Leukodystrophy.

Investigating the role of POLR3 in oligodendrocyte-lineage development and myelination in vivo: Translational disease models and proof-of-concept for using in vivo gene therapy to treat POLR3-related Hypomyelinating Leukodystrophy.
研究 POLR3 在体内少突胶质细胞谱系发育和髓鞘形成中的作用：转化疾病模型和使用体内基因疗法治疗 POLR3 相关的低髓鞘性脑白质营养不良的概念验证。
批准号：
415860
负责人：
Michell-Robinson Mackenzie A
金额：
$10.93万
依托单位：
McGill University
依托单位国家：
加拿大
项目类别：
Studentship Programs
财政年份：
2019
资助国家：
加拿大
项目状态：
已结题
起止时间：
2019-11-01 至 2022-11-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=415860&lang=en
关键词：
Adeno-Associated Virus Animal Models Of Cns Disease In Vitro Models Of Cns Disease Lineage Development Myelination Neurodegenerative Disease Neurodevelopment Pediatric Disease Translational Research White Matter Disease

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Leukodystrophies are a group of rare genetic diseases affecting brain development that collectively occur in 1:7500 individuals. Children with leukodystrophy grow normally until they develop neurological signs, especially movement problems. Based on medic
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