PUBLICATION

Inhibition of Lysosome Membrane Recycling Causes Accumulation of Gangliosides that Contribute to Neurodegeneration

Authors
Boutry, M., Branchu, J., Lustremant, C., Pujol, C., Pernelle, J., Matusiak, R., Seyer, A., Poirel, M., Chu-Van, E., Pierga, A., Dobrenis, K., Puech, J.P., Caillaud, C., Durr, A., Brice, A., Colsch, B., Mochel, F., El Hachimi, K.H., Stevanin, G., Darios, F.
ID
ZDB-PUB-180628-15
Date
2018
Source
Cell Reports   23: 3813-3826 (Journal)
Registered Authors
Keywords
autophagic lysosome recovery, autophagy, glycosphingolipids, induced pluripotent stem cells, knockout, lipid metabolism, lysosomes, membrane trafficking, neurodegenerative disease, organoids
MeSH Terms
  • Animals
  • Autophagy/drug effects
  • Female
  • Gangliosides/metabolism*
  • Glutamic Acid/pharmacology
  • Humans
  • Induced Pluripotent Stem Cells/cytology
  • Induced Pluripotent Stem Cells/metabolism
  • Intracellular Membranes/metabolism*
  • Lysosomes/drug effects
  • Lysosomes/metabolism*
  • Mice
  • Mice, Knockout
  • Neurons/cytology
  • Neurons/metabolism
  • Proteins/genetics
  • Proteins/metabolism
  • Spastic Paraplegia, Hereditary/metabolism
  • Spastic Paraplegia, Hereditary/pathology
  • Zebrafish/metabolism
  • Zebrafish Proteins/deficiency
  • Zebrafish Proteins/genetics
PubMed
29949766 Full text @ Cell Rep.
Abstract
Lysosome membrane recycling occurs at the end of the autophagic pathway and requires proteins that are mostly encoded by genes mutated in neurodegenerative diseases. However, its implication in neuronal death is still unclear. Here, we show that spatacsin, which is required for lysosome recycling and whose loss of function leads to hereditary spastic paraplegia 11 (SPG11), promotes clearance of gangliosides from lysosomes in mouse and human SPG11 models. We demonstrate that spatacsin acts downstream of clathrin and recruits dynamin to allow lysosome membrane recycling and clearance of gangliosides from lysosomes. Gangliosides contributed to the accumulation of autophagy markers in lysosomes and to neuronal death. In contrast, decreasing ganglioside synthesis prevented neurodegeneration and improved motor phenotype in a SPG11 zebrafish model. Our work reveals how inhibition of lysosome membrane recycling leads to the deleterious accumulation of gangliosides, linking lysosome recycling to neurodegeneration.
Genes / Markers
Figures
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Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping