Evaluating CFTR-function and response to new CFTR modulator drugs in non-cystic fibrosis patients with CFTR-related lung disease

评估患有 CFTR 相关肺部疾病的非囊性纤维化患者的 CFTR 功能和对新型 CFTR 调节药物的反应

• 批准号: 435003
• 负责人: Gonska Tanja
• 金额: $ 56.79万
• 依托单位: Hospital for Sick Children (Toronto)
• 依托单位国家: 加拿大
• 项目类别: Operating Grants
• 财政年份: 2020
• 资助国家: 加拿大
• 起止时间: 2020-10-01 至 2025-10-01
• 项目状态: 未结题
• 来源: https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=435003&lang=en
• 关键词: Cftr Modulator Drugs; Cftr-Related Disease; Cystic Fibrosis Newborn Screening; Nasal Cells

Defects in the Cystic Fibrosis gene (CFTR) cause Cystic Fibrosis (CF), a multi-organ disease with progressive lung disease as the main cause for early mortality. Next to classic CF, there are patients presenting later in life with chronic lung disease. Ex

囊性纤维化基因 (CFTR) 缺陷会导致囊性纤维化 (CF)，这是一种以进行性肺病为早期死亡主要原因的多器官疾病。除了经典的 CF 之外，还有一些患者在晚年出现慢性肺病。前任