Increased phosphorylation of HexM improves lysosomal uptake and potential for managing GM2 gangliosidoses

Graeme Benzie, Kristen Bouma,Taylor Battellino, Steven Cooper,Rick Hemming,Wafa Kammouni, Lin Liu, Cuong Do,Mazdak Khajehpour,Helene Perreault,Stuart Kornfeld,Barbara Triggs-Raine,Brian L. Mark

BBA advances（2022）

引用 0|浏览2

暂无评分

摘要

•Tay-Sachs and Sandhoff disease are caused by loss of β-hexosaminidase A (HexA).•Scalable production of a stable version of HexA, known as HexM, provides potential for an enzyme replacement therapy.•Phosphorylation of HexM by an engineered GlcNAc-1-phosphotransferase significantly enhances cellular uptake.

查看译文

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