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

Improved efficacy of a next-generation ERT in murine Pompe disease

下一代 ERT 治疗小鼠庞贝病的疗效提高

Su Xu, Yi Lun, Michelle Frascella, Anadina Garcia, Rebecca Soska, Anju Nair, Abdul S Ponery, Adriane Schilling, Jessie Feng, Steven Tuske, Maria Cecilia Della Valle, José A Martina, Evelyn Ralston, Russell Gotschall, Kenneth J Valenzano, Rosa Puertollano, Hung V Do, Nina Raben, Richie Khanna

Coformulation of a Novel Human α-Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry Mice

新型人类 α-半乳糖苷酶 A 与药理伴侣 AT1001 的共配制可改善法布里病小鼠的底物减少

Su Xu, Yi Lun, Nastry Brignol, Rick Hamler, Adriane Schilling, Michelle Frascella, Sean Sullivan, Robert E Boyd, Kate Chang, Rebecca Soska, Anadina Garcia, Jessie Feng, Hidehito Yasukawa, Carole Shardlow, Alison Churchill, Amol Ketkar, Nicola Robertson, Masahito Miyamoto, Kazutoshi Mihara, Elfrida R

The pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe disease

药理学伴侣 AT2220 可增加突变酸性 α-葡萄糖苷酶的特异性活性和溶酶体递送,并促进庞贝病转基因小鼠模型中的糖原减少

Richie Khanna, Allan C Powe Jr, Yi Lun, Rebecca Soska, Jessie Feng, Rohini Dhulipala, Michelle Frascella, Anadina Garcia, Lee J Pellegrino, Su Xu, Nastry Brignol, Matthew J Toth, Hung V Do, David J Lockhart, Brandon A Wustman, Kenneth J Valenzano