Gmppb-mutant mice exhibit dystroglycanopathy symptoms that are rescued with GSK3β inhibition or AAV-mediated GMPPB gene replacement

Z Ziwei Fu T Tongchao Wang C Chenyang Zhang (Department of Biopharmaceutics, Zhejiang Provincial Engineering Research Center of New Technologies and Applications for Targeted Therapy of Major Diseases, College of Life Science and Medicine, Zhejiang Sci-Tech University) T Tianyu Qi Y Yanyan Chen J Ju Yang H Hua Yang (State Key Laboratory of Natural Medicines, School of Pharmacy, China Pharmaceutical University, 24 Tong Jia Xiang, Nanjing 210009, China) B Bing Yan (Key Laboratory of Biomacromolecules, Institute of Biophysics, Chinese Academy of Sciences) B Baoming Gong W Weiqiao Lu S Sushan Luo Y Ying Liu L Lei Sun H Hao Jiang B Bo Chen Z Zhao Zhang X Xiuping Liu Y Yuxiang Wang (Key Laboratory of Photochemical Conversion and Optoelectronic Materials & CAS-HKU Joint Laboratory on New Materials)