CAG-targeted brain-permeable therapy tested in biallelic humanized polyQ mouse models.
在双等位基因人源化polyQ小鼠模型中测试了CAG靶向脑渗透性疗法
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2025.102496
Surdyka Magdalena, Kalinowska-PoÅka Å»aneta, Niewiadomska-Cimicka Anna, Jesion Ewelina, Fiszer Agnieszka, Singer-Mikosch Elisabeth, Fievet Lorraine, Przybyl Lukasz, Caron Nicholas S, Hayden Michael R, Nguyen Huu Phuc, Trottier Yvon, Figiel Maciej
