A Compact Base Editor Rescues AATD-associated Liver and Lung Disease in Mouse Models
Jenny Gao, Nathan Bamidele, Debora Pires-Ferreira, Chunwei Zheng, April Destefano, Haoyang Cheng, Qiushi Tang, Yueying Cao, Jun Xie, Guangping Gao, Alisha M. Gruntman, Erik J. Sontheimer, Terence R. Flotte, Wen Xue
This paper demonstrates that a compact ABE corrects the PiZ mutation in AATD mouse models, reducing liver disease and improving lung function.
Molecular Therapy · Aug 28, 2025Peer-reviewedBioengineering