In vivo Base-Editing Corrects Metabolic Defects in Glycogen Storage Disease Type-Ia
Y. Aratyn-Schaus,I. Arnaoutova,D. LeBoeuf,T. P. Fernandez,M. Packer,L. Zhang,L. Cheng,G. Lung,R. Dutko,F. Musenge,K. Ramanan,S. E. Smith,J. Decker,R. Yang,J. R. Dorkin,D. Chen,G. Ciaramella,F. M. Gregoire,J. Y. Chou
