A Pentasaccharide for Monitoring Pharmacodynamic Response to Gene Therapy in GM1 Gangliosidosis

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

EBioMedicine（2023）

引用 1|浏览40

暂无评分

关键词

Gene therapy,GM1 gangliosidosis,Pentasaccharide,Pharmacodynamic biomarker

AI 理解论文

溯源树

样例

生成溯源树，研究论文发展脉络

Chat Paper

正在生成论文摘要