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: 479527
• 负责人: Bernard Geneviève
• 金额: $ 72.45万
• 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
• 起止时间: 2023-03-01 至 2028-03-01
• 项目状态: 未结题
• 来源: https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=479527&lang=en
• 关键词: Adeno-Associated Virus; Cellular Models; Gene Therapy; Mouse Models; Myelination; Oligodendrocyte Progenitor Cells And Oligodendrocytes; Polr3-Related Leukodystrophy; Rna Polymerase Iii; Small Molecule Therapy

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

脑白质营养不良是一组罕见的遗传性神经系统疾病，影响以前健康的儿童，他们在数月至数年内逐渐残疾，最终死于早期死亡。大多数这些癌症没有治愈的治疗方法。