日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Novel promoters drive therapeutic transgene expression and evade transgene-specific immune responses in a mouse model of Pompe disease

新型启动子驱动治疗性转基因表达,并在庞贝病小鼠模型中逃避转基因特异性免疫反应

Sun, Baodong; Yi, Haiqing; Han, Sang-Oh; Li, Songtao; Eisner, William; Brooks, Elizabeth D; Bacon, Rebecca; Koeberl, Dwight D

Gain-of-function ANXA11 mutation cause late-onset ALS with aberrant protein aggregation, neuroinflammation and autophagy impairment

获得功能性 ANXA11 突变导致晚发型 ALS,伴有异常蛋白质聚集、神经炎症和自噬障碍

Qing Liu, Ye Sun, Baodong He, Haodong Chen, Lijing Wang, Gaojie Wang, Kang Zhang, Ximeng Zhao, Xinzhe Zhang, Dongchao Shen, Xue Zhang, Liying Cui

High-potency MyoAAV capsids enhanced skeletal muscle correction in a mouse model of GSD IIIa

高活性MyoAAV衣壳增强了GSD IIIa小鼠模型的骨骼肌矫正效果

Liao, Kuo-An; Han, Sang-Oh; Barzi, Mercedes; Yi, Haiqing; Eisner, William; Bissig-Choisat, Beatrice; Bissig, Karl-Dimiter; Sun, Baodong

Long-Term Correction of Murine Glycogen Storage Disease Type III by AAV-Mediated Gene Therapy Using an Immunotolerizing Dual Promoter to Express Bacterial Pullulanase

利用免疫耐受双启动子表达细菌普鲁兰酶,通过腺相关病毒介导的基因疗法长期纠正小鼠糖原贮积症III型

Liao, Kuo-An; Lim, Jeong-A; Choi, Su Jin; Yi, Haiqing; Sun, Baodong

Gene therapy for glycogen storage diseases

糖原贮积症的基因疗法

Koeberl, Dwight D; Koch, Rebecca L; Lim, Jeong-A; Brooks, Elizabeth D; Arnson, Benjamin D; Sun, Baodong; Kishnani, Priya S

Characterization of liver GSD IX γ2 pathophysiology in a novel Phkg2(-/-) mouse model

利用新型 Phkg2(-/-) 小鼠模型表征肝脏 GSD IX γ2 的病理生理学

Gibson, Rebecca A; Lim, Jeong-A; Choi, Su Jin; Flores, Leticia; Clinton, Lani; Bali, Deeksha; Young, Sarah; Asokan, Aravind; Sun, Baodong; Kishnani, Priya S

An emerging phenotype of central nervous system involvement in Pompe disease: from bench to bedside and beyond

庞贝病中枢神经系统受累的新兴表型:从实验室到临床及其他

Korlimarla, Aditi; Lim, Jeong-A; Kishnani, Priya S; Sun, Baodong

IL-37 is associated with osteoarthritis disease activity and suppresses proinflammatory cytokines production in synovial cells

IL-37与骨关节炎疾病活动性相关,并抑制滑膜细胞中促炎细胞因子的产生。

Ding, Liping; Hong, Xiaoping; Sun, Baodong; Huang, Qin; Wang, Xiaoqi; Liu, Xiaokai; Li, Lingyun; Huang, Zhong; Liu, Dongzhou

Alglucosidase alfa treatment alleviates liver disease in a mouse model of glycogen storage disease type IV

阿糖苷酶α治疗可缓解IV型糖原贮积症小鼠模型中的肝脏疾病。

Yi, Haiqing; Gao, Fengqin; Austin, Stephanie; Kishnani, Priya S; Sun, Baodong

A Modified Enzymatic Method for Measurement of Glycogen Content in Glycogen Storage Disease Type IV

一种改良的酶法测定IV型糖原贮积症中糖原含量的方法

Yi, Haiqing; Zhang, Quan; Yang, Chunyu; Kishnani, Priya S; Sun, Baodong