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Designing a toolbox of paradigmatic treatments for a targeted molecular medicine approach to autosomal-recessive ataxias

Designing a toolbox of paradigmatic treatments for a targeted molecular medicine approach to autosomal-recessive ataxias
设计针对常染色体隐性共济失调的靶向分子医学方法的范例治疗工具箱
批准号：
430840
负责人：
Brais Bernard
金额：
$16.39万
依托单位：
McGill University
依托单位国家：
加拿大
项目类别：
Operating Grants
财政年份：
2020
资助国家：
加拿大
项目状态：
已结题
起止时间：
2020-06-01 至 2023-06-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=430840&lang=en
关键词：
Ataxia Biomarker Discovery Biomarker Validation Drug Development Drug Validation Proteomics Transcriptomics Transgenic Mice Models

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中文摘要
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英文摘要
Autosomal-recessive cerebellar ataxia (ARCA) is a heterogeneous group of rare neurodegenerative genetic diseases that share the common hallmark of progressive damage to parts of the nervous system that coordinate movement, such as the cerebellum. Despite
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Characterization of the newly uncovered internationally prevalent GAA triplet expansion late-onset SCA27B ataxia that is more common in Quebec: from improved phenotyping, genotyping to molecular pathological understanding
  • 批准号：
    478132
  • 项目类别：
    Operating Grants
  • 资助金额：
    $26.75万
  • 财政年份：
    2023
  • 负责人：
    Brais Bernard
  • 依托单位：
    McGill University
PROSPAX: an integrated multimodal progression chart in spastic ataxias
  • 批准号：
    406801
  • 项目类别：
    Operating Grants
  • 资助金额：
    $16.35万
  • 财政年份：
    2019
  • 负责人：
    Brais Bernard
  • 依托单位：
    McGill University
PREPARE: Preparing for therapies in autosomal recessive ataxias
New emerging team on Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay (ARSACS): from models to treatment strategies
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