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: Universit Laval
• 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
• 起止时间: 2019-03-01 至 2024-03-01
• 项目状态: 已结题
• 来源: https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=400052&lang=en
• 关键词: Crispr/Cas9; Friedreich Ataxia,; Gaa Repeat Deletion; Yg8sr

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

弗里德赖希共济失调（FRDA）是最常见的遗传性共济失调。这是一种隐性疾病，即，父母双方都必须是突变基因的携带者。然而，它们不是症状。只有继承了两个变异基因拷贝的孩子才能