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Many neurodegenerative diseases are characterized by the accumulation of undegradable molecules in cells or at extracellular sites in the brain. One such family of diseases is the lysosomal storage disorders, which result from defects in various aspects of lysosomal function. Until recently, there was little prospect of treating storage diseases involving the CNS. However, recent progress has been made in understanding these conditions and in translating the findings into experimental therapies. We review the developments in this field and discuss the similarities in pathological features between these diseases and some more common neurodegenerative disorders.

Original publication




Journal article


Nat Rev Neurosci

Publication Date





713 - 725


Animals, Brain, Calcium, G(M1) Ganglioside, Homeostasis, Humans, Lysosomal Storage Diseases, Membrane Microdomains, Models, Biological, Protein Transport, Sphingolipids