Activation of endogenous full-length utrophin by MyoAAV-UA as a therapeutic approach for Duchenne muscular dystrophy.
利用 MyoAAV-UA 激活内源性全长肌营养不良蛋白作为治疗杜氏肌营养不良症的一种方法
期刊:Nature Communications
影响因子:15.7
doi:10.1038/s41467-025-57831-5
Wu Ruo, Li Peng, Xiao Puhao, Zhang Shu, Wang Xiaopeng, Liu Jie, Sun Wenjie, Chang Yue, Ai Xiuyi, Chen Lijiao, Zhuo Yan, Wang Jiaojian, Wang Zhengbo, Li Shangang, Li Yuanyuan, Ji Weizhi, Guo Wenting, Wu Shiwen, Chen Yongchang
