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Removal of the GAA repeat with the CRISPR/Cas9 system in Friedreich patient cells and in the YG8sR mouse model."

Removal of the GAA repeat with the CRISPR/Cas9 system in Friedreich patient cells and in the YG8sR mouse model."
在 Friedreich 患者细胞和 YG8sR 小鼠模型中使用 CRISPR/Cas9 系统去除 GAA 重复序列。”
批准号:
400052
负责人:
Tremblay Jacques P
金额:
$54.89万
依托单位:
依托单位国家:
加拿大
项目类别:
Operating Grants
财政年份:
2019
资助国家:
加拿大
项目状态:
已结题
起止时间:
2019-03-01 至 2024-03-01

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中文摘要
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英文摘要
Friedreich Ataxia (FRDA) is the most common form of hereditary ataxia. This is a recessive disease, i.e., both parents have to be carriers of the mutated gene. However, they are not symptomatic. Only the child who inherits two copies of the mutated gene w
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