GLA-modified RNA treatment lowers GB3 levels in iPSC-derived cardiomyocytes from Fabry-affected individuals
GLA修饰的RNA治疗可降低法布里病患者iPSC来源心肌细胞中的GB3水平
期刊:American Journal of Human Genetics
影响因子:8.1
doi:10.1016/j.ajhg.2023.07.013
Ter Huurne, Menno; Parker, Benjamin L; Liu, Ning Qing; Qian, Elizabeth Ling; Vivien, Celine; Karavendzas, Kathy; Mills, Richard J; Saville, Jennifer T; Abu-Bonsrah, Dad; Wise, Andrea F; Hudson, James E; Talbot, Andrew S; Finn, Patrick F; Martini, Paolo G V; Fuller, Maria; Ricardo, Sharon D; Watt, Kevin I; Nicholls, Kathy M; Porrello, Enzo R; Elliott, David A