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

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

Sphingosine-1-phosphate lyase insufficiency syndrome (SPLIS) is a rare metabolic disorder caused by inactivating mutations in sphingosine-1-phosphate lyase 1 (SGPL1), which is required for the final step of sphingolipid metabolism. SPLIS features include steroid-resistant nephrotic syndrome and impa...

Full description

Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: American Society for Clinical investigation 2021-04-01
Series:JCI Insight
Subjects:
Metabolism
Therapeutics
Online Access:https://doi.org/10.1172/jci.insight.145936

Internet

https://doi.org/10.1172/jci.insight.145936

Similar Items