Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates
系统性 mRNA 疗法治疗法布里病:野生型小鼠、法布里病小鼠模型和野生型非人类灵长类动物的临床前研究
期刊:American Journal of Human Genetics
影响因子:8.1
doi:10.1016/j.ajhg.2019.02.003
Xuling Zhu, Ling Yin, Matt Theisen, Jenny Zhuo, Summar Siddiqui, Becca Levy, Vladimir Presnyak, Andrea Frassetto, Jaclyn Milton, Timothy Salerno, Kerry E Benenato, Joe Milano, Andy Lynn, Staci Sabnis, Kristine Burke, Gilles Besin, Christine M Lukacs, Lin T Guey, Patrick F Finn, Paolo G V Martini