BioengineeringMolecular TherapyPeer-reviewed
A Compact Base Editor Rescues AATD-associated Liver and Lung Disease in Mouse Models
Jenny Gao, Nathan Bamidele, Debora Pires-Ferreira…·Wen Xue
This paper demonstrates that a compact ABE corrects the PiZ mutation in AATD mouse models, reducing liver disease and improving lung function.
4 citations