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Target intelligence / Profile preview
GM2 Ganglioside (GM2)
- Target
- GM2
- Molecular classification
- Glycosphingolipid, Ganglioside
01
Overview
GM2 ganglioside is a structurally defined glycosphingolipid crucial for normal neural function but pathogenic when accumulated due to enzymatic deficiencies. It serves both physiological roles at cellular membranes and pathological roles when dysregulated or targeted by immunotherapies against certain cancers. Accumulation of GM2 occurs due to deficiencies in enzymes responsible for its degradation, leading to lysosomal storage disorders. GM2 also acts as an antigen expressed on various human cancer cells.
02
Mechanism of action
GM2-containing vaccines elicit antibody production.
03
Biological functions
Cell signalingNeuronal developmentImmune modulationRegulation of cell proliferationRegulation of cell differentiationRegulation of apoptosisAntigen presentation
04
Disease associations
Tay-Sachs diseaseSandhoff diseaseAB variant diseaseCancerNeurodegenerative disease
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