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

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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