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RNA polymerase III-related leukodystrophy: Understanding disease pathogenesis and developing novel therapeutic approaches using murine models

RNA polymerase III-related leukodystrophy: Understanding disease pathogenesis and developing novel therapeutic approaches using murine models
RNA 聚合酶 III 相关脑白质营养不良：了解疾病发病机制并利用小鼠模型开发新的治疗方法
批准号：
472825
负责人：
Bernard Geneviève
金额：
$7.29万
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：
已结题
起止时间：
2022-09-01 至 2023-09-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=472825&lang=en
关键词：
Adeno-Associated Virus Cellular Models Gene Therapy Mouse Models Myelination Oligodendrocyte Progenitor Cells Oligodendrocytes Polr3-Related Leukodystrophy Rna Polymerase Iii Small Molecule Therapy

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英文摘要
Leukodystrophies are a group of rare genetic neurological diseases affecting previously healthy children, who become progressively disabled over months to years, eventually succumbing to early death. No curative treatment is available for most of these ch
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RNA polymerase III-related leukodystrophy: Understanding disease pathogenesis and developing novel therapeutic approaches using murine models
Unraveling the molecular basis of novel forms of hypomyelinating leukodystrophies
Canadian Leukodystrophy Conference for Patients and Families: Current and Novel therapies
Unraveling the molecular basis of novel forms of hypomyelinating leukodystrophies
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