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Target intelligence / Profile preview
GM2 (Ganglioside)
- Molecular classification
- Glycosphingolipid, Ganglioside
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Overview
GM2 (ganglioside) is a glycosphingolipid primarily found in neuronal cell membranes. It plays roles in cell signaling, neuronal development, and immune responses. Its accumulation due to genetic defects in catabolic enzymes leads to severe neurodegenerative disorders called GM2 gangliosidoses (e.g., Tay-Sachs, Sandhoff). GM2 is also expressed on some cancer cells, making it a target for antibody-based immunotherapies.
02
Mechanism of action
Antibody-mediated cell death/inhibition of metastasis (for anti-GM2 antibodies)
03
Biological functions
Cell signaling modulationNeuronal developmentImmune responseCell proliferation regulationCell differentiation regulationApoptosis regulation
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Disease associations
Neurodegenerative disease (GM2 gangliosidoses)Cancer (tumor antigen)
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Safety considerations
Potential for off-target effects (though GM2 expression is relatively restricted)Immune-related adverse events with antibody therapies
06
Interacting drugs
Anti-GM2 antibodies
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