Using a patient-derived fibroblast ciliation assay to perform high throughput drug screen and candidate therapeutic compound validation for Bardet-Biedl syndrome (BBS)

使用患者来源的成纤维细胞纤毛检测对 Bardet-Biedl 综合征 (BBS) 进行高通量药物筛选和候选治疗化合物验证

• 批准号: 449358
• 负责人: Pearson Alexander L
• 金额: $ 1.27万
• 依托单位: University of Toronto
• 依托单位国家: 加拿大
• 项目类别: Studentship Programs
• 财政年份: 2020
• 资助国家: 加拿大
• 起止时间: 2020-12-01 至 2021-12-01
• 项目状态: 已结题
• 来源: https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=449358&lang=en
• 关键词: Bardet-Biedl Syndrome; Blindness; Ciliopathy; Fibroblasts; Genetic Disease; High Throughput Drug Screen; Pharmaceutical; Therapeutics

Bardet-Biedl syndrome (BBS) is a rare autosomal recessive genetic disorder caused by defective structure and/or function of the primary cilia. BBS is characterized by developmental anomalies, obesity, and a progressive retinal degeneration. Most BBS patie

Bardet-Biedl综合征（BBS）是由原发性纤毛的有缺陷和/或功能引起的罕见常染色体隐性遗传疾病。 BBS的特征是发育异常，肥胖和进行性视网膜变性。大多数bbs patie