Cookies on this website
We use cookies to ensure that we give you the best experience on our website. If you click 'Continue' we'll assume that you are happy to receive all cookies and you won't see this message again. Click 'Find out more' for information on how to change your cookie settings.

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

DOI

10.1038/nrn1725

Type

Journal article

Journal

Nat Rev Neurosci

Publication Date

09/2005

Volume

6

Pages

713 - 725

Keywords

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