Target intelligence / Profile preview

Ganglioside GM2

Molecular classification
Glycosphingolipid, Monosialo-ganglioside, Lipid
01

Overview

Ganglioside GM2 is a glycosphingolipid found in the outer leaflet of cell membranes. It plays roles in cell signaling, neuronal development, immune responses, and is implicated in lysosomal storage disorders and cancer. As a tumor-associated antigen, it is a target for cancer immunotherapy.

Other names
Tay–Sachs gangliosideβ-D-GalNAc-(1→4)-[α-Neu5Ac-(2→3)]-β-D-Gal-(1→4)-β-D-Glc-(1↔1)-N-octadecanoylsphingosine
02

Mechanism of action

Antibodies targeting GM2 have shown anti-tumor effects both experimentally and clinically.

03

Biological functions

Membrane organizationCell signalingNeuronal developmentImmune modulationCell adhesionSignal transduction
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Disease associations

Tay-Sachs diseaseSandhoff diseaseAB variant diseaseCancerNeurodegenerative disease
05

Safety considerations

Potential off-target effects if not specifically targeted in immunotherapy; however, antibodies targeting GM2 have shown minimal toxicity related to off-target effects on healthy tissue in clinical trials.

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