Base-editing Corrects Metabolic Abnormalities in a Humanized Mouse Model for Glycogen Storage Disease Type-Ia
Irina Arnaoutova,Yvonne Aratyn-Schaus,Lisa Zhang,Michael S. Packer,Hung-Dar Chen,Cheol Lee,Sudeep Gautam,Francine M. Gregoire,Dominique Leboeuf, Steven Boule,Thomas P. Fernandez,Victoria Huang,Lo- Cheng,Genesis Lung, Brianna Bannister,Jeremy Decker,Thomas Leete, Lan S. Shuang, Caroline Bock, Prachi Kothiyal,Phil Grayson, Ka W. Mok, Jeffrey J. Quinn,Lauren Young,Luis Barrera,Giuseppe Ciaramella,Brian C. Mansfield,Janice Y. Chou NATURE COMMUNICATIONS（2024）
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Glycogen Storage Diseases,Glucocerebrosidase Mutations
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