A systemically deliverable lipid-conjugated siRNA targeting DUX4 as an facioscapulohumeral muscular dystrophy therapeutic.
一种可系统递送的脂质偶联 siRNA，靶向 DUX4，作为面肩肱型肌营养不良症的治疗方法
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2025.101513
Daman Katelyn, Yan Jing, Biscans Annabelle, Echeverria Dimas, Shmushkovich Taisia, Wolfson Alexey, Alterman Julia F, Khvorova Anastasia, Emerson Charles P Jr
