Efficacy of AAV9-mediated SGPL1 gene transfer in a mouse model of S1P lyase insufficiency syndrome

por Zhao, Piming, Tassew, Gizachew B., Lee, Joanna Y., Oskouian, Babak, Muñoz, Denise P., Hodgin, Jeffrey B., Watson, Gordon L., Tang, Felicia, Wang, Jen-Yeu, Luo, Jinghui, Yang, Yingbao, King, Sarah, Krauss, Ronald M., Keller, Nancy, Saba, Julie D.
Publicado 2021