A systemically delivered AAV-CFTR gene therapy for cystic fibrosis
Lindsey W. Plasschaert, Cian Stutz, Ester Otarola, Lionello Ruggeri, Rachel Valdez Misiolek, Arthur G. Nuccio, Jianyu Shang, Rayman Choo-Wing, Bruck Taddese, Arnaud Decock, Catherine Quigley, Katie Kubek-Luck, Isabelle Warnant, Daher Ibrahim Aibo, Maria Chiara Magnifico, , Amy Lin
This paper develops a systemic AAV-CFTR gene therapy approach for cystic fibrosis, demonstrating functional CFTR expression and lung transduction in non-human primates.
bioRxiv · Mar 20, 2025 · PreprintGenetics