Efficacy of AAV9-mediated SGPL1 gene transfer in a mouse model of S1P lyase insufficiency syndrome
AAV9 介导的 SGPL1 基因转移对 S1P 裂解酶不足综合征小鼠模型的疗效
期刊:JCI Insight
影响因子:6.3
doi:10.1172/jci.insight.145936
Piming Zhao, Gizachew B Tassew, Joanna Y Lee, Babak Oskouian, Denise P Muñoz, Jeffrey B Hodgin, Gordon L Watson, Felicia Tang, Jen-Yeu Wang, Jinghui Luo, Yingbao Yang, Sarah King, Ronald M Krauss, Nancy Keller, Julie D Saba