Ashley L. Cooney

This paper develops novel AAV capsids with improved transduction efficiency for airway epithelial cells, reducing required doses and enhancing therapeutic potential for respiratory diseases.

This study tests lentiviral-mediated ABCA3 gene complementation in cell lines with specific pathogenic variants, showing partial rescue of cellular functions and differential effects based on variant type.

This paper shows that in utero ivacaftor treatment rescues multiple organ defects in CFTR-G551D pigs, improving pancreatic, gallbladder, and vas deferens function.

This study shows that lentiviral vector transduction of airway basal cells does not impair their differentiation or function, suggesting potential for long-term gene therapy in lung diseases.