Summaries by Inventor Doray, Balraj
Lysosomal Enzyme GlcNAc-1-phosphotransferase with Enhanced Ability to Phosphorylate Lysosomal Enzymes
Doray, Balraj ; Kornfeld, Stuart ; Lee, Wang ; Liu, Lin
T-016569
Doray, Balraj ; Kornfeld, Stuart ; Lee, Wang ; Liu, Lin
T-016569
— 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…