A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis
用于监测 GM1 神经节苷脂沉积症基因治疗药效学反应的五糖
期刊:EBioMedicine
影响因子:9.7
doi:10.1016/j.ebiom.2023.104627
Pamela Kell, Rohini Sidhu, Mingxing Qian, Sonali Mishra, Elena-Raluca Nicoli, Precilla D'Souza, Cynthia J Tifft, Amanda L Gross, Heather L Gray-Edwards, Douglas R Martin, Miguel Sena-Esteves, Dennis J Dietzen, Manmilan Singh, Jingqin Luo, Jean E Schaffer, Daniel S Ory, Xuntian Jiang