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

AAV-based gene therapy prevents and halts the progression of dilated cardiomyopathy in a mouse model of phosphoglucomutase 1 deficiency (PGM1-CDG)

基于 AAV 的基因治疗可预防和阻止磷酸葡糖变位酶 1 缺乏症 (PGM1-CDG) 小鼠模型中扩张型心肌病的进展

Bijina Balakrishnan, Ruqaiah Altassan, Rohit Budhraja, Willisa Liou, Arielle Lupo, Sarah Bryant, Anastasiya Mankouski, Silvia Radenkovic, Graeme J Preston, Akhilesh Pandey, Sihem Boudina, Tamas Kozicz, Eva Morava, Kent Lai

The Metabolic Map into the Pathomechanism and Treatment of PGM1-CDG

PGM1-CDG 发病机制和治疗的代谢图

Silvia Radenkovic, Matthew J Bird, Tim L Emmerzaal, Sunnie Y Wong, Catarina Felgueira, Kyle M Stiers, Leila Sabbagh, Nastassja Himmelreich, Gernot Poschet, Petra Windmolders, Jan Verheijen, Peter Witters, Ruqaiah Altassan, Tomas Honzik, Tuba F Eminoglu, Phillip M James, Andrew C Edmondson, Jozef Her