Identifying the role of airway stem cells in maintaining lentiviral mediated gene expression for cystic fibrosis lung disease

囊性纤维化（CF）是一种进行性、慢性和衰弱性遗传疾病，可导致肺部疾病的早期死亡。虽然已经出现了针对大约2000种CF突变中的一些的药物治疗，但只有一种基因疗法可以治疗所有的CF突变类型。该项目将评估一种新的气道预处理是否能提高慢病毒基因转移的水平和持续时间，以及有效的长期基因表达是否是导导常驻气道基底干细胞的结果。

Cystic fibrosis (CF) is a progressive, chronic and debilitating genetic disease that casuses early death from lung disease. Although drug treatments for a few of the ~2000 CF mutations have appeared, only a genetic therapy can treat all CF mutation types. This project will assess whether a novel airway pre-treatment improves the level and duration of lentiviral gene transfer, and whether effective long-term gene expression is the result of transducing resident airway basal stem cells.