建立和新型的人类翻译模型，以推进囊性纤维化研究、药物发现，并优化 CFTR 靶向治疗药物。
Established and novel human translational models to advance cystic fibrosis research, drug discovery, and optimize CFTR-targeting therapeutics.
机构信息
Marsico Lung Institute and Cystic Fibrosis Research Center, School of Medicine, University of North Carolina, Chapel Hill, NC 27599, USA.
Marsico Lung Institute and Cystic Fibrosis Research Center, School of Medicine, University of North Carolina, Chapel Hill, NC 27599, USA; Department of Pediatrics, Division of Pediatric Pulmonology, School of Medicine, University of North Carolina School of Medicine, Chapel Hill, NC 27599, USA; Department of Cell Biology and Physiology, School of Medicine, University of North Carolina, Chapel Hill, NC 27599, USA.
出版信息
Curr Opin Pharmacol. 2022 Jun;64:102210. doi: 10.1016/j.coph.2022.102210. Epub 2022 Apr 21.
To find a cure for cystic fibrosis, there has been tremendous progress in the development of treatments that target the basic defect in the protein channel, CFTR. However, 10% of cystic fibrosis patients have rare CFTR mutations that are still without an approved CFTR-targeting drug. To identify relevant therapies for these patients, culture models using nasal, bronchial, and rectal tissue from individual patients allow functional, biochemical, and cellular detection of drug-rescued CFTR. Additionally, novel systems such as induced pluripotent stem cell-derived models are utilized to characterize CFTR mutations and identify treatments. State-of-the-art translational models were instrumental for CFTR modulator development and may become important for gene-based drug discovery and other novel therapeutic strategies.
为了找到囊性纤维化的治疗方法，在针对蛋白通道 CFTR 基本缺陷的治疗方法的开发方面已经取得了巨大的进展。然而，10%的囊性纤维化患者存在罕见的 CFTR 突变，仍然没有批准的针对 CFTR 的药物。为了为这些患者找到相关的治疗方法，使用来自个体患者的鼻、支气管和直肠组织的培养模型允许对药物挽救的 CFTR 进行功能、生化和细胞检测。此外，还利用诱导多能干细胞衍生的模型等新型系统来表征 CFTR 突变并确定治疗方法。最先进的转化模型对于 CFTR 调节剂的开发至关重要，并且可能对基于基因的药物发现和其他新型治疗策略变得重要。
Zotero 插件