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Rationally designed ribozymes switched-on by nucleotide repeat extensions as potential tools of genetic therapy for repeat expansion disorders.

Rationally designed ribozymes switched-on by nucleotide repeat extensions as potential tools of genetic therapy for repeat expansion disorders.
合理设计的核酶通过核苷酸重复延伸开启,作为重复扩张疾病基因治疗的潜在工具。
批准号:
464593
负责人:
Rouleau Guy A
金额:
$7.29万
依托单位:
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:
2022-03-01 至 2023-03-01

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中文摘要
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英文摘要
Over 40 diseases, including incurable and fatal neurodegenerative diseases such as ALS, are caused by the expansion of repetitive sequences in the genome. Beyond a certain size, these expansions cause problems in the cells and the body, leading to disease
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Rationally designed ribozymes switched-on by nucleotide repeat extensions as potential tools of genetic therapy for repeat expansion disorders.
  • 批准号:
    479481
  • 项目类别:
    Operating Grants
  • 资助金额:
    $77.74万
  • 财政年份:
    2023
  • 负责人:
    Rouleau Guy A
  • 依托单位:
Advancing our understanding of essential tremor genetics and biology
  • 批准号:
    483432
  • 项目类别:
    Operating Grants
  • 资助金额:
    $7.29万
  • 财政年份:
    2023
  • 负责人:
    Rouleau Guy A
  • 依托单位:
Advancing our understanding of essential tremor genetics and biology
  • 批准号:
    489526
  • 项目类别:
    Operating Grants
  • 资助金额:
    $63.25万
  • 财政年份:
    2023
  • 负责人:
    Rouleau Guy A
  • 依托单位:
Rationally designed ribozymes switched-on by nucleotide repeat extensions as potential tools of genetic therapy for repeat expansion disorders.
  • 批准号:
    472596
  • 项目类别:
    Operating Grants
  • 资助金额:
    $7.29万
  • 财政年份:
    2022
  • 负责人:
    Rouleau Guy A
  • 依托单位:
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