Secondary accumulation of gangliosides in lysosomal storage disorders

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162 Scopus citations


Glycosphingolipids (GSLs) known as gangliosides have been documented to accumulate in a wide range of lysosomal storage disorders, including those with and without primary defects in ganglioside degradation. The same two gangliosides, GM2 and GM3, are often found elevated in diseased neurons whereas in normal mature neurons both are essentially undetectable. Altered expression of these two gangliosides does not appear to result solely from cellularity changes or gliosis since immunocytochemical studies show that both GM2 and GM3 reside in vesicular structures within affected neurons. Elevated expression of one of these gangliosides (GM2) has also been found to closely correlate with the growth of ectopic dendrites on susceptible neurons, a phenomenon that uniquely characterizes many lysosomal diseases. Understanding the precise role of the endosomal-lysosomal system in the overall homeostatic control of GSL expression in neurons can be expected to provide key insight into both the function of gangliosides and the pathogenic mechanisms underlying lysosomal disease.

Original languageEnglish (US)
Pages (from-to)433-444
Number of pages12
JournalSeminars in Cell and Developmental Biology
Issue number4
StatePublished - Aug 2004


  • Axon
  • Axonal spheroid
  • Cholesterol
  • Dendrite
  • Endosome
  • Glycosphingolipid
  • Lysosome
  • Raft

ASJC Scopus subject areas

  • Developmental Biology
  • Cell Biology


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