Translating cystic fibrosis gene-addition therapy for use in human lungs: Demonstrating efficacy, safety and scalability

像Kalydeco这样昂贵的药物可以改善囊性纤维化(CF)的肺健康，但仅在某些患者中使用。呼吸道基因加成疗法可以治疗任何患者。我们成功的呼吸道基因治疗方法直到我们最近研制出患有CF的大鼠后才能在CF肺中进行测试。这个项目将精确地将我们的基因疗法定位于肺部区域，使用包括我们革命性的x射线方法在内的技术来评估其有效性和安全性，并在人类大小的绵羊肺中测试其潜力。

Expensive drugs like Kalydeco can improve cystic fibrosis (CF) lung health, but only in some patients. Airway gene-addition therapy could treat any patient. Our successful airway gene therapy method could not be tested in CF lungs until our recent development of rats with CF. This project will precisely-target our gene therapy to lung regions, assess the effectiveness and safety with techniques including our revolutionary X-ray methods, and test its potential in the human-size lungs of sheep