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）肺部健康，但仅适用于某些患者。气道基因添加疗法可以治疗任何患者。我们成功的气道基因治疗方法无法在CF肺中进行测试，直到我们最近开发出患有CF的大鼠。该项目将精确地将我们的基因治疗靶向肺部区域，通过包括我们革命性的X射线方法在内的技术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