| Gene Symbol | G3BP1 |
| Full Name | Ras-GTPase-Activating Protein-Binding Protein 1 |
| Chromosome | 5q33.1 |
| Protein Type | Signaling Protein |
| Target Class | Signaling Protein |
| Function | SG Nucleation: G3BP1 acts as a scaffold for SG assembly, recruiting other SG components |
| Mechanism of Action | G3BP1 inhibitors would prevent stress granule assembly and stabilization by disrupting the interaction between G3BP1 and its binding partners, potentially reducing pathological aggregation of TDP-43 and FUS proteins. This mechanism could alleviate neurodegeneration in conditions characterized by aberrant stress granule formation and RNA metabolism dysfunction. |
| Primary Expression | Ubiquitous, high in brain (neurons, glia) |
| Druggability | Medium (0.47) |
| Clinical Stage | Phase II |
| Molecular Weight | ~52 kDa |
| Pathways | Apoptosis, Autophagy, Er Stress, Immune Response, Mtor |
| UniProt ID | A0A7I2V3C4 |
| NCBI Gene ID | 9973 |
| Ensembl ID | ENSG00000130830 |
| GeneCards | G3BP1 |
| Human Protein Atlas | G3BP1 |
| Associated Diseases | AD, ALI, ALS, AMI, Alzheimer |
| Known Drugs/Compounds | axonal mRNA, Intracellular Free Rna, trehalose, EGCG, rapamycin |
| Interactions | TOLLIP, SQSTM1, NDP52, TAX1BP1, FUS, PEX3 |
| SciDEX Target | View Target Profile (7 clinical trials) |
| SciDEX Hypotheses | Phase-Separated Organelle Targeting Stress Granule Phase Separation Modulators RNA Granule Nucleation Site Modulation (+1 more) |
| KG Connections | 1219 knowledge graph edges |
| Databases | GeneCardsHPASTRING |