Lysosomal Hydrolases
Inborn Errors of Metabolism
Lysosomes
Glucose Transporters
Protein Import into the Peroxisomes
Delivery Pathways to the Lysosome
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In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
Published on: December 20, 2017
José A Martina1, Nina Raben1, Rosa Puertollano1
1Cell and Developmental Biology Center, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
Lysosomal storage diseases (LSDs) involve inherited metabolic disorders where lysosomes accumulate undegraded material. This review covers protein functions, pathogenesis, and current therapies for LSDs.
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