Patrick L. Sinn

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 paper identifies two host pathways, epithelial-to-mesenchymal transition and live cell extrusion, that facilitate the detachment of MeV-infected cell clusters from human airway epithelia, enhancing viral transmission.

This paper develops a PCD pig model with DNAI1 mutations and characterizes its respiratory and sinus disease features, showing similarities to human PCD.

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.

This study compares rAAV production methods using different host cells, finding chemically and functionally distinct vectors with differences in PTMs, methylation, and potency.