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Allele-specific lowering of mutant polyQ proteins as treatment for Huntington disease, spinocerebellar ataxia type 3 and spinocerebellar ataxia type 7

Allele-specific lowering of mutant polyQ proteins as treatment for Huntington disease, spinocerebellar ataxia type 3 and spinocerebellar ataxia type 7
等位基因特异性降低突变 PolyQ 蛋白作为亨廷顿病、脊髓小脑共济失调 3 型和脊髓小脑共济失调 7 型的治疗
批准号：
373043
负责人：
Hayden Michael R
金额：
$32.29万
依托单位：
BC Children's Hospital Research Institute
依托单位国家：
加拿大
项目类别：
Operating Grants
财政年份：
2017
资助国家：
加拿大
项目状态：
已结题
起止时间：
2017-06-01 至 2020-06-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=373043&lang=en
关键词：
Animal Models Antisense Oligonucleotides Crispr/Cas9 Gene Silencing Huntington Disease Polyglutamine Diseases Sca3 Sca7 Sirna

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中文摘要
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英文摘要
Polyglutamine (polyQ) diseases are a group of monogenic, autosomal dominant neurodegenerative disorders caused by a polyQ encoding CAG triplet repeat expansion in the disease causing gene with pathogenic consequences. The most prevalent polyQ disorders ar
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Novel Approaches Directed to Prevention and Treatment of Huntington Disease
Canada Research Chair Tier 1
  • 批准号：
    315426
  • 项目类别：
  • 资助金额：
    $101.99万
  • 财政年份：
    2014
  • 负责人：
    Hayden Michael R
  • 依托单位：
    University of British Columbia
Novel approaches to modulate key targets in the pathogenesis of Huntington Disease
Novel approaches to modulate key targets in the pathogenesis of Huntington Disease
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