Development of an AAV-delivered microRNA gene therapy for myotonic dystrophy type 1
开发用于治疗1型强直性肌营养不良症的AAV递送microRNA基因疗法
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.08.050
Tomassy, Giulio S; Fan, Wei; Cao, Shuwen; Luo, Zhengyu; Magli, Alessandro; Zhang, Tracy; Boyle, Kristen; Jackson, Robert; Richards, Brenda; Liu, Dongyu; Rapaport, Franck; Khader, Shameer; Pfister, Edith L; O'Riordan, Catherine; Frederick, Amy; Sullivan, Jennifer; Cao, James; Nageswara Rao, Kollu; Assaf, Basel T; Goulet, Martin; Mueller, Christian