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Golgi-specific brefeldin A-resistance guanine nucleotide exchange factor 1 (GBF1)
- Target
- GBF1
- Molecular classification
- Enzyme (specifically a guanine nucleotide exchange factor, or GEF)
01
Overview
Golgi-specific brefeldin A-resistance guanine nucleotide exchange factor 1 (GBF1) is a high-molecular-weight guanine nucleotide exchange factor (GEF) essential for the activation of ADP-ribosylation factor (Arf) family GTPases, primarily Arf1, Arf4, and Arf5. It is predominantly cytosolic but cycles rapidly onto and off Golgi membranes, initiating vesicular transport via COPI-coated vesicles and maintaining Golgi structure. Structurally, GBF1 contains a central catalytic Sec7 domain and several regulatory domains (DCB, HUS, HDS1-3), enabling multiple protein-protein and membrane interactions. Pharmacologically, GBF1 is inhibited by drugs like brefeldin A and the more specific golgicide A, which block nucleotide exchange and arrest vesicular traffic, causing Golgi disassembly and ER swelling. GBF1's pivotal role in trafficking and its involvement in viral replication, along with links to hereditary neurodegenerative diseases, make it an important therapeutic and research target.
- Other names
- GBF1KIAA0248BFA-resistant GEF 1ARF1GEFCMT2GGCMTDI2CMTDIA
02
Mechanism of action
Competitive inhibition of Sec7 domain (drugs bind the interface and block the conformational change necessary for Arf1 GDP-GTP exchange)
03
Biological functions
Regulation of vesicle trafficking (COPI vesicle transport, ER-Golgi and intra-Golgi transport)Activation of Arf family GTPases (Arf1, Arf4, Arf5)Maintenance of Golgi structure and homeostasisMembrane association and cycling (between cytosol and Golgi)Involvement in lipid droplet and mitochondrial functionRole in clathrin-independent endocytosis and chemotaxis
04
Disease associations
Viral infection/replication (essential host factor for RNA virus replication)Potential roles in neurodegenerative disease (due to linkage with Charcot-Marie-Tooth disease subtypes: CMT2GG, CMTDI2, CMTDIA)Golgi-related disorders and possibly cancer (due to vesicular trafficking disruption and Golgi disassembly)
05
Safety considerations
Disruption of vesicular trafficking (leads to Golgi disassembly, ER swelling, possible cell death)Essential role in cellular/organismal function (systemic inhibition can be cytotoxic)
06
Interacting drugs
Brefeldin A (BFA; inhibits GBF1, BIG1, BIG2)
1 more in the full profile.
07
Biomarkers
None widely established; patient selection uses indirect markers (e.g., Golgi disassembly or ER swelling upon BFA/GCA treatment may indicate GBF1 inhibition)
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