Lysosomal Enzyme GlcNAc-1-phosphotransferase with Enhanced Ability to Phosphorylate Lysosomal Enzymes

by | Oct 22, 2019 | Doray, Balraj, Kornfeld, Stuart, Lee, Wang, Liu, Lin

— Lysosomal storage disorders result in severe, multi-system dysfunction and frequently, early death. Currently, a number of lysosomal storage disorders are treated with enzyme replacement therapy (ERT), which requires periodic infusions of the deficient enzyme. However, in a number of instances the…