GABRG1

Summary

GABRG1 (gamma-aminobutyric acid type A receptor subunit gamma1, HGNC:4086) is a protein-coding gene on chromosome 4p12, encoding Gamma-aminobutyric acid receptor subunit gamma-1 (Q8N1C3). Gamma subunit of the heteropentameric ligand-gated chloride channel gated by gamma-aminobutyric acid (GABA), a major inhibitory neurotransmitter in the brain.

The protein encoded by this gene belongs to the ligand-gated ionic channel family. It is an integral membrane protein and plays an important role in inhibiting neurotransmission by binding to the benzodiazepine receptor and opening an integral chloride channel. This gene is clustered with three other family members on chromosome 4.

Source: NCBI Gene 2565 — RefSeq curated summary.

At a glance

• Gene–disease (curated): genetic developmental and epileptic encephalopathy (Limited, GenCC)
• GWAS associations: 6
• Clinical variants (ClinVar): 64 total
• Druggable target: yes — 15 molecules with ChEMBL bioactivity
• MANE Select transcript: NM_173536

Identifiers

Gene identifiers

Field	Value
HGNC ID	HGNC:4086
Approved symbol	GABRG1
Name	gamma-aminobutyric acid type A receptor subunit gamma1
Location	4p12
Locus type	gene with protein product
Status	Approved
Ensembl gene	ENSG00000163285
Ensembl biotype	protein_coding
OMIM	137166
Entrez	2565

Gene structure

Transcript identifiers

Ensembl transcripts: 2 — 2 protein_coding

ENST00000295452, ENST00000964875

RefSeq mRNA: 1 — MANE Select: NM_173536 NM_173536

CCDS: CCDS3470

Canonical transcript exons

ENST00000295452 — 9 exons

Exon	Start	End
ENSE00001073015	46065364	46065584
ENSE00001073017	46058485	46058622
ENSE00001073019	46097201	46097349
ENSE00001073021	46123810	46124054
ENSE00001073022	46051424	46051638
ENSE00001073024	46064441	46064523
ENSE00001073026	46083986	46084053
ENSE00001073028	46058217	46058369
ENSE00001258742	46035769	46041254

Expression profiles

Bgee: expression breadth broad, 88 present calls, max score 92.57.

FANTOM5 (CAGE): breadth tissue_specific, TPM avg 5.8550 / max 645.9126, expressed in 109 samples.

FANTOM5 promoters (4 alternative TSS)

Promoter ID	TPM avg	Samples expressed
51995	3.7866	108
51997	1.5585	94
51998	0.3200	85
51996	0.1899	77

Top tissues by expression

226 total, by Bgee expression score (0-100, higher = more expressed):

Tissue	Anatomy ID	Expression score	Quality
medial globus pallidus	UBERON:0002477	92.57	gold quality
globus pallidus	UBERON:0001875	91.95	gold quality
endothelial cell	CL:0000115	90.50	gold quality
lateral globus pallidus	UBERON:0002476	89.74	gold quality
Brodmann (1909) area 23	UBERON:0013554	89.28	gold quality
entorhinal cortex	UBERON:0002728	88.74	gold quality
caudate nucleus	UBERON:0001873	88.21	gold quality
nucleus accumbens	UBERON:0001882	87.96	gold quality
temporal lobe	UBERON:0001871	86.92	gold quality
amygdala	UBERON:0001876	86.65	gold quality
Brodmann (1909) area 46	UBERON:0006483	86.38	gold quality
hypothalamus	UBERON:0001898	86.20	gold quality
superior frontal gyrus	UBERON:0002661	86.18	gold quality
postcentral gyrus	UBERON:0002581	85.89	gold quality
putamen	UBERON:0001874	85.37	gold quality
superior vestibular nucleus	UBERON:0007227	85.29	gold quality
primary visual cortex	UBERON:0002436	84.63	gold quality
Ammon’s horn	UBERON:0001954	84.48	gold quality
parietal lobe	UBERON:0001872	84.37	gold quality
dorsolateral prefrontal cortex	UBERON:0009834	83.65	gold quality
Brodmann (1909) area 9	UBERON:0013540	83.46	gold quality
occipital lobe	UBERON:0002021	83.44	gold quality
right frontal lobe	UBERON:0002810	83.11	gold quality
anterior cingulate cortex	UBERON:0009835	83.05	gold quality
cerebral cortex	UBERON:0000956	82.98	gold quality
substantia nigra pars reticulata	UBERON:0001966	82.90	gold quality
substantia nigra	UBERON:0002038	82.84	gold quality
corpus callosum	UBERON:0002336	82.73	gold quality
medulla oblongata	UBERON:0001896	82.61	gold quality
frontal cortex	UBERON:0001870	82.27	gold quality

Single-cell (SCXA)

Detected in 1 experiment(s), a significant marker in 1.

Experiment	Marker?	Max mean expression
E-ANND-3	yes	4.71

Regulation

Is transcription factor: no

miRNA regulators (miRDB)

229 targeting GABRG1, top 30 by miRDB confidence (max_score; target_count = how many genes the miRNA targets in total — lower means more specific):

miRNA	Max score	Avg score	miRNA target_count
HSA-MIR-5011-5P	100.00	83.46	5820
HSA-MIR-190A-3P	100.00	80.35	5520
HSA-MIR-98-3P	100.00	74.08	3907
HSA-MIR-5692A	100.00	74.40	6850
HSA-LET-7A-3P	100.00	74.03	3932
HSA-LET-7B-3P	100.00	74.08	3913
HSA-LET-7F-1-3P	100.00	74.02	3928
HSA-MIR-3646	100.00	73.56	5283
HSA-MIR-1277-5P	100.00	73.95	5056
HSA-MIR-3613-3P	100.00	76.36	7965
HSA-MIR-3064-3P	100.00	70.09	1254
HSA-MIR-4668-3P	100.00	68.74	2635
HSA-MIR-3924	100.00	72.09	2394
HSA-MIR-3163	100.00	77.23	8605
HSA-MIR-4262	100.00	73.26	3931
HSA-MIR-8485	100.00	77.57	4731
HSA-MIR-4776-3P	100.00	68.73	1340
HSA-MIR-3662	99.99	73.82	5684
HSA-MIR-548C-3P	99.99	74.01	7587
HSA-MIR-186-5P	99.99	70.83	3707
HSA-MIR-4282	99.99	75.36	6408
HSA-MIR-371B-5P	99.99	75.34	4759
HSA-MIR-3667-3P	99.99	67.17	1636
HSA-MIR-4775	99.98	75.00	6394
HSA-MIR-4789-5P	99.98	70.76	2721
HSA-MIR-373-5P	99.98	75.36	4753
HSA-MIR-616-5P	99.98	75.58	4775
HSA-LET-7F-2-3P	99.98	70.98	2588
HSA-MIR-4803	99.98	71.99	3117
HSA-MIR-1185-1-3P	99.98	71.04	2593

Literature-anchored findings (GeneRIF, showing 11)

• Logistic regression analysis indicated that genetic elements in the GABRG1 haplotype block likely contribute to alcohol dependence risk in an additive manner. (PMID:17507911)
• study of patterns of htSNPs & linkage disequilibrium (LD)in 6 populations; LD extended from most of the GABRA2 gene through the GABRG1 locus in the same GABAA cluster region suggesting possible association of these genes with alcohol dependence (PMID:17976953)
• results of the present study suggest that hippocampal neurons expressing GABAAgamma receptor subunits resist the progression of neurofibrillary degeneration in Alzheimer disease hippocampus (PMID:19019179)
• A single nucleotide polymorphism of the GABRG1 gene (rs1497571) is associated with level of response to alcohol and drinking patterns. (PMID:19426171)
• An intronic single nucleotide polymorphism (SNP) (rs17536211) in GABRG1 shows the strongest statistical evidence of association of recovery from eating disorders. (PMID:21750581)
• we genotyped 13 single nucleotide polymorphisms spanning GABRG1 and GABRA2 in African Americans with alcohol dependence (PMID:21919924)
• A significant GABRG1 genotype influences self-administration of alcohol and GABAergic manipulation. (PMID:22817768)
• Switching the intracellular domains of the gamma2 and gamma1 subunits substantiated this inference. Because this region determines post-synaptic localization, we hypothesize that GABAARs containing gamma1 and gamma2 use different mechanisms for synaptic clustering. (PMID:24425869)
• rs1497577 not significantly associated with adaptation to subjective responses to alcohol (PMID:26087834)
• Steady-state activation of the high-affinity isoform of the alpha4beta2delta GABAA receptor. (PMID:31690811)
• The strongest association for alcohol use disorder (rs3756007) was located in GABRA2, the strongest association for antisocial behavior (rs11941860) was located in GABRG1, and the interaction term yielded top associations in GABRA2 (rs2119183) and the intergenic region between GABRA2 and GABRG1 (rs536599). (PMID:31790348)

Cross-species orthologs

3 orthologs

Organism	Symbol	Gene ID
danio_rerio	gabrg1	ENSDARG00000101995
mus_musculus	Gabrg1	ENSMUSG00000001260
rattus_norvegicus	Gabrg1	ENSRNOG00000002360

Paralogs (45): GABRA3 (ENSG00000011677), GABRA1 (ENSG00000022355), CHRNA3 (ENSG00000080644), GABRP (ENSG00000094755), CHRNA4 (ENSG00000101204), GLRA2 (ENSG00000101958), GABRE (ENSG00000102287), CHRNE (ENSG00000108556), GABRA4 (ENSG00000109158), GLRB (ENSG00000109738), GABRR2 (ENSG00000111886), GABRG2 (ENSG00000113327), CHRNB4 (ENSG00000117971), CHRNA2 (ENSG00000120903), CHRNA10 (ENSG00000129749), CHRND (ENSG00000135902), CHRNA1 (ENSG00000138435), GLRA3 (ENSG00000145451), GABRA6 (ENSG00000145863), GABRB2 (ENSG00000145864), GLRA1 (ENSG00000145888), GABRR1 (ENSG00000146276), CHRNB3 (ENSG00000147432), CHRNA6 (ENSG00000147434), HTR3B (ENSG00000149305), GABRA2 (ENSG00000151834), CHRNB2 (ENSG00000160716), GABRB1 (ENSG00000163288), GABRB3 (ENSG00000166206), CHRFAM7A (ENSG00000166664), HTR3A (ENSG00000166736), CHRNA5 (ENSG00000169684), CHRNB1 (ENSG00000170175), CHRNA9 (ENSG00000174343), CHRNA7 (ENSG00000175344), HTR3C (ENSG00000178084), GABRG3 (ENSG00000182256), GABRR3 (ENSG00000183185), HTR3E (ENSG00000186038), HTR3D (ENSG00000186090)

Protein

Protein identifiers

Gamma-aminobutyric acid receptor subunit gamma-1 — Q8N1C3 (reviewed: Q8N1C3)

Alternative names: GABA(A) receptor subunit gamma-1

All UniProt accessions (1): Q8N1C3

UniProt curated annotations — full annotation on UniProt →

Function. Gamma subunit of the heteropentameric ligand-gated chloride channel gated by gamma-aminobutyric acid (GABA), a major inhibitory neurotransmitter in the brain. GABA-gated chloride channels, also named GABA(A) receptors (GABAAR), consist of five subunits arranged around a central pore and contain GABA active binding site(s) located at the alpha and beta subunit interface(s). When activated by GABA, GABAARs selectively allow the flow of chloride anions across the cell membrane down their electrochemical gradient. Chloride influx into the postsynaptic neuron following GABAAR opening decreases the neuron ability to generate a new action potential, thereby reducing nerve transmission.

Subunit / interactions. Heteropentamer, formed by a combination of alpha (GABRA1-6), beta (GABRB1-3), gamma (GABRG1-3), delta (GABRD), epsilon (GABRE), rho (GABRR1-3), pi (GABRP) and theta (GABRQ) chains, each subunit exhibiting distinct physiological and pharmacological properties.

Subcellular location. Postsynaptic cell membrane. Cell membrane.

Post-translational modifications. May be palmitoylated.

Domain organisation. GABAARs subunits share a common topological structure: a peptide sequence made up of a long extracellular N-terminal, four transmembrane domains, intracellular or cytoplasmic domain located between the third and the fourth transmembrane domains.

Similarity. Belongs to the ligand-gated ion channel (TC 1.A.9) family. Gamma-aminobutyric acid receptor (TC 1.A.9.5) subfamily. GABRG1 sub-subfamily.

RefSeq proteins (1): NP_775807* (*=MANE)

Domains & families (InterPro)

ID	Name	Type
IPR005437	GABRG-1/4	Family
IPR005438	GABBAg1_rcpt	Family
IPR006028	GABAA/Glycine_rcpt	Family
IPR006029	Neurotrans-gated_channel_TM	Domain
IPR006201	Neur_channel	Family
IPR006202	Neur_chan_lig-bd	Domain
IPR018000	Neurotransmitter_ion_chnl_CS	Conserved_site
IPR036719	Neuro-gated_channel_TM_sf	Homologous_superfamily
IPR036734	Neur_chan_lig-bd_sf	Homologous_superfamily
IPR038050	Neuro_actylchol_rec	Homologous_superfamily

Pfam: PF02931, PF02932

Catalyzed reactions (Rhea), 1 shown:

• chloride(in) = chloride(out) (RHEA:29823)

UniProt features (15 total): topological domain 4, transmembrane region 4, glycosylation site 3, signal peptide 1, chain 1, disulfide bond 1, sequence variant 1

Structure

Experimental structures (PDB)

0 structures.

Predicted structure (AlphaFold)

Model	pLDDT	Fraction very-high
AF-Q8N1C3-F1	79.06	0.61

Functional residue map

Curated UniProt residues grouped by drug-discovery relevance — catalytic, ligand-binding, modification, and mutation-validated positions. Source: UniProtKB sequence features.

Disulfide bonds (1): 188–202

Glycosylation sites (3): 50, 127, 245

Function

Pathways and Gene Ontology

Reactome pathways

0 pathways

MSigDB gene sets: 167 (showing top): GSE18804_BRAIN_VS_COLON_TUMORAL_MACROPHAGE_DN, GOBP_SYNAPSE_ASSEMBLY, AAGCCAT_MIR135A_MIR135B, GOBP_INORGANIC_ANION_TRANSPORT, GOBP_GAMMA_AMINOBUTYRIC_ACID_SIGNALING_PATHWAY, LHX3_01, GOBP_CELL_CELL_SIGNALING, chr4p12, GOBP_CELL_JUNCTION_ORGANIZATION, GOBP_CHLORIDE_TRANSPORT, KEGG_NEUROACTIVE_LIGAND_RECEPTOR_INTERACTION, TGACATY_UNKNOWN, GOBP_SYNAPTIC_SIGNALING, AACTTT_UNKNOWN, GOBP_CELL_JUNCTION_ASSEMBLY

GO Biological Process (8): gamma-aminobutyric acid signaling pathway (GO:0007214), synaptic transmission, GABAergic (GO:0051932), chloride transmembrane transport (GO:1902476), inhibitory synapse assembly (GO:1904862), monoatomic ion transport (GO:0006811), chloride transport (GO:0006821), chemical synaptic transmission (GO:0007268), monoatomic ion transmembrane transport (GO:0034220)

GO Molecular Function (9): GABA-A receptor activity (GO:0004890), GABA-gated chloride ion channel activity (GO:0022851), GABA receptor binding (GO:0050811), transmembrane signaling receptor activity (GO:0004888), monoatomic ion channel activity (GO:0005216), extracellular ligand-gated monoatomic ion channel activity (GO:0005230), chloride channel activity (GO:0005254), protein binding (GO:0005515), benzodiazepine receptor activity (GO:0008503)

GO Cellular Component (9): plasma membrane (GO:0005886), dendrite membrane (GO:0032590), chloride channel complex (GO:0034707), postsynaptic membrane (GO:0045211), postsynapse (GO:0098794), GABA-A receptor complex (GO:1902711), membrane (GO:0016020), signaling receptor complex (GO:0043235), synapse (GO:0045202)

GO top-level categories

Rollup of top GO terms by namespace:

Category	Terms
GABA receptor activity	2
cellular anatomical structure	2
cell-cell signaling	1
chemical synaptic transmission	1
chloride transport	1
monoatomic anion transmembrane transport	1
synapse assembly	1
transport	1
monoatomic anion transport	1
inorganic anion transport	1
anterograde trans-synaptic signaling	1
monoatomic ion transport	1
transmembrane transport	1
chloride channel activity	1
transmitter-gated monoatomic ion channel activity	1
ligand-gated monoatomic anion channel activity	1
signaling receptor binding	1
signaling receptor activity	1
monoatomic ion transmembrane transporter activity	1
channel activity	1
ligand-gated monoatomic ion channel activity	1
monoatomic anion channel activity	1
chloride transmembrane transporter activity	1
binding	1
neurotransmitter receptor activity	1
membrane	1
cell periphery	1
dendrite	1
neuron projection membrane	1
monoatomic ion channel complex	1
synaptic membrane	1
postsynapse	1
synapse	1
GABA receptor complex	1
protein-containing complex	1
cell junction	1

Protein interactions and networks

STRING

2154 interactions, top by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
GABRG1	GABRB1	P18505	635
GABRG1	GABRA2	P47869	491
GABRG1	ACADM	P11310	480
GABRG1	GABBR2	O75899	467
GABRG1	GABRA4	P48169	456
GABRG1	PGM1	P36871	423
GABRG1	TTC12	Q9H892	413
GABRG1	NMUR1	Q9HB89	410
GABRG1	GAD1	Q99259	407
GABRG1	OPRD1	P41143	401
GABRG1	CHRM2	P08172	398
GABRG1	SLC6A1	P30531	388
GABRG1	DRD2	P14416	387
GABRG1	ACOT13	Q9NPJ3	381
GABRG1	GRIK1	P39086	381

IntAct

7 interactions, top by confidence:

A	B	Type	Score
GABRG1	OPTN	psi-mi:“MI:0915”(physical association)	0.560
GABRG2	GPAA1	psi-mi:“MI:0914”(association)	0.530
ECE1	GABRG1	psi-mi:“MI:0915”(physical association)	0.370
GABRG2	B4GALT5	psi-mi:“MI:0914”(association)	0.350

BioGRID (4): GABRG1 (Affinity Capture-MS), GABRG1 (Affinity Capture-MS), SHANK2 (Cross-Linking-MS (XL-MS)), GABRG1 (Cross-Linking-MS (XL-MS))

ESM2 similar proteins: D1LYT2, O94925, P08219, P08220, P0C2W5, P10063, P10064, P14867, P15431, P16305, P18505, P18507, P18508, P19019, P19150, P19969, P20236, P21548, P22300, P22723, P23574, P23576, P24045, P26048, P26049, P27681, P28472, P28473, P30191, P31644, P34903, P47869, P47870, P50571, P62812, P62813, P63079, P63080, P63137, P63138

Diamond homologs: A8MPY1, D1LYT2, F1R8P4, G5EBR3, O00591, O09028, O14764, O18276, O75311, O93430, P07727, P08219, P08220, P0C2W5, P10063, P10064, P14867, P15431, P16305, P18505, P18506, P18507, P18508, P19019, P19150, P19969, P20236, P20237, P20781, P21548, P22300, P22723, P22771, P22933, P23415, P23416, P23574, P23576, P24045, P24046

SIGNOR signaling

0 interactions.

Disease & clinical

Clinical variants and AI predictions

ClinVar

64 variants total. Per-class counts are floors (≥ shown; pagination cap):

Classification	Count (floor)
Pathogenic	0
Likely pathogenic	0
Uncertain significance	59
Likely benign	0
Benign	0

Top pathogenic / likely-pathogenic (0)

SpliceAI

1458 predictions. Top by Δscore:

Variant	Effect	Δscore
4:46051591:T:TA	donor_gain	1.0000
4:46064432:GTTAC:G	donor_loss	1.0000
4:46064433:TTACT:T	donor_loss	1.0000
4:46064434:TACTT:T	donor_loss	1.0000
4:46064435:ACT:A	donor_loss	1.0000
4:46064436:CTT:C	donor_loss	1.0000
4:46064437:TTACA:T	donor_loss	1.0000
4:46064438:TACAG:T	donor_loss	1.0000
4:46064439:A:AC	donor_gain	1.0000
4:46064439:ACA:A	donor_loss	1.0000
4:46064439:ACAG:A	donor_gain	1.0000
4:46064440:C:CC	donor_gain	1.0000
4:46064440:CA:C	donor_gain	1.0000
4:46064440:CAG:C	donor_gain	1.0000
4:46064440:CAGC:C	donor_gain	1.0000
4:46064440:CAGCT:C	donor_gain	1.0000
4:46064520:CAAT:C	acceptor_gain	1.0000
4:46064523:TC:T	acceptor_loss	1.0000
4:46064524:C:CC	acceptor_gain	1.0000
4:46064525:T:A	acceptor_loss	1.0000
4:46064528:T:C	acceptor_gain	1.0000
4:46083980:ACTT:A	donor_loss	1.0000
4:46083981:CTT:C	donor_loss	1.0000
4:46083982:TTAC:T	donor_loss	1.0000
4:46083983:TAC:T	donor_loss	1.0000
4:46083984:A:AC	donor_gain	1.0000
4:46083984:A:T	donor_loss	1.0000
4:46083984:AC:A	donor_gain	1.0000
4:46083985:C:CC	donor_gain	1.0000
4:46083985:C:CG	donor_loss	1.0000

AlphaMissense

3079 scored. Top likely-pathogenic:

Variant	Protein change	am_pathogenicity
4:46051535:A:C	C340W	1.000
4:46051536:C:T	C340Y	1.000
4:46051537:A:G	C340R	1.000
4:46051608:A:G	L316P	1.000
4:46051623:A:G	L311P	1.000
4:46051623:A:T	L311Q	1.000
4:46051626:A:T	V310D	1.000
4:46058217:C:G	G306R	1.000
4:46058227:T:A	R302S	1.000
4:46058227:T:G	R302S	1.000
4:46058228:C:G	R302T	1.000
4:46058257:A:C	F292L	1.000
4:46058257:A:T	F292L	1.000
4:46058259:A:G	F292L	1.000
4:46058290:G:C	C281W	1.000
4:46058294:G:C	P280R	1.000
4:46058294:G:T	P280Q	1.000
4:46058329:T:A	R268S	1.000
4:46058329:T:G	R268S	1.000
4:46058330:C:A	R268I	1.000
4:46058330:C:G	R268T	1.000
4:46058336:A:G	L266P	1.000
4:46058534:A:C	F238L	1.000
4:46058534:A:T	F238L	1.000
4:46058535:A:C	F238C	1.000
4:46058536:A:G	F238L	1.000
4:46058588:C:A	W220C	1.000
4:46058588:C:G	W220C	1.000
4:46058590:A:G	W220R	1.000
4:46058590:A:T	W220R	1.000

dbSNP variants (sampled 300 via entrez): RS1000002138 (4:46042536 T>C), RS1000002176 (4:46043014 AAGAG>A,AAG), RS1000003028 (4:46080136 A>G), RS1000037817 (4:46036788 A>C), RS1000080894 (4:46118651 C>G), RS1000163685 (4:46089352 A>G), RS1000166661 (4:46045234 A>G), RS1000210963 (4:46058351 G>A,C,T), RS1000221378 (4:46042266 A>G), RS1000228142 (4:46087079 AT>A,ATT), RS1000242468 (4:46120280 C>T), RS1000271356 (4:46112464 C>T), RS1000321667 (4:46125831 C>T), RS1000321682 (4:46048579 T>C), RS1000365286 (4:46053546 A>C)

Disease associations

OMIM: gene MIM:137166 | disease phenotypes:

GenCC curated gene-disease

Disease	Classification	Inheritance
genetic developmental and epileptic encephalopathy	Limited	Autosomal dominant

Mondo (2): prostate cancer (MONDO:0008315), genetic developmental and epileptic encephalopathy (MONDO:0100062)

Orphanet (1): Familial prostate cancer (Orphanet:1331)

HPO phenotypes

0 total (0 of 0 shown, HPO-id order):

GWAS associations

6 associations (top):

Study	Trait	p-value
GCST004904_19	Body mass index	1.000000e-17
GCST004904_70	Body mass index	3.000000e-48
GCST006462_11	Uterine fibroids	2.000000e-10
GCST006462_12	Uterine fibroids	2.000000e-11
GCST006462_13	Uterine fibroids	3.000000e-11
GCST006462_14	Uterine fibroids	3.000000e-11

EFO canonical traits (1, from GWAS)

EFO ID	Trait name
EFO:0004340	body mass index

MeSH disease descriptors (1)

Descriptor	Name	Tree numbers
D011471	Prostatic Neoplasms	C04.588.945.440.770; C12.100.500.260.750; C12.100.500.565.625; C12.200.294.260.750; C12.200.294.565.625; C12.200.758.409.750; C12.900.619.750

Drugs & pharmacology

Drug and pharmacology data

Is drug target: yes

ChEMBL targets (2): CHEMBL2093872 (PROTEIN COMPLEX GROUP), CHEMBL2109243 (PROTEIN COMPLEX GROUP)

Molecules with ChEMBL bioactivity

15 molecules (phase ≥1), by development phase (incl. off-target/promiscuous compounds). Patent mentions across the top 20 by phase: 442,649 (via chembl_molecule»patent_compound — counts attach to the compound, not the gene–compound relationship, so off-target/promiscuous molecules can dominate).

Molecule	Name	Phase	Patents
CHEMBL1082407	ENZALUTAMIDE	4	9,652
CHEMBL12	DIAZEPAM	4	92,281
CHEMBL1544	LIOTHYRONINE	4	23,700
CHEMBL1568698	GANAXOLONE	4	1,657
CHEMBL207538	BREXANOLONE	4	1,585
CHEMBL3183409	APALUTAMIDE	4	4,076
CHEMBL407	FLUMAZENIL	4	7,150
CHEMBL452	CLONAZEPAM	4	33,297
CHEMBL268254	DELORAZEPAM	2	1,308
CHEMBL275638	FLAVONE	2	88,985
CHEMBL287631	PROGABIDE	2	3,853
CHEMBL454095	ABECARNIL	2	566
CHEMBL8260	BAICALEIN	2	8,592
CHEMBL273481	MUSCIMOL	1	5,759
CHEMBL96	GAMMA-AMINOBUTYRIC ACID	1	160,188

PharmGKB: 1 entry (VIP=true, CPIC=false)

GtoPdb / IUPHAR curated pharmacology

(IUPHAR/BPS Guide to Pharmacology — expert-curated)

Target class: lgic — GABA_A receptors

Binding affinities (BindingDB)

191 measured of 201 human assays (202 total across all organisms); most potent 50 below. Values come from heterogeneous assays and are not directly comparable.

Ligand	Measure	Value	Patent
3-[7-chloro-8-(trifluoromethyl)-4H-[1,2,4]triazolo[4,3-a][1,4]benzodiazepin-6-yl]-4-fluoro-phenol	KI	0.27 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
3-(7,8-dichloro-1-methyl-4H-[1,2,4]triazolo[4,3-a][1,4]benzodiazepin-6-yl)-4-fluoro-phenol	KI	0.36 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
4-fluoro-3-[(4S)-7,8-dichloro-1,4-dimethyl-4H-[1,2,4]triazolo[4,3-a][1,4]benzodiazepin-6-yl]phenol	KI	0.38 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
(5S)-8,9-dichloro-7-(2-fluoro-5-hydroxy-phenyl)-5-methyl-5H-pyrimido[1,2-a][1,4]benzodiazepin-3-one	KI	0.39 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
8,9-dichloro-7-(2-fluoro-5-hydroxy-phenyl)-5H-pyrimido[1,2-a][1,4]benzodiazepin-3-one	KI	0.39 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
3-(7,8-dichloro-1-methyl-4H-[1,2,4]triazolo[4,3-a][1,4]benzodiazepin-6-yl)-2,4-difluoro-phenol	KI	0.4 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
6-chloro-5-(2-fluoro-5-hydroxy-phenyl)-1-methyl-7-(trifluoromethyl)-3H-1,4-benzodiazepin-2-one	KI	0.44 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
6,7-dichloro-5-(2,6-difluoro-3-hydroxy-phenyl)-1-methyl-3H-1,4-benzodiazepin-2-one	KI	0.46 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
Halcion	KI	0.68 nM
8,9-dichloro-7-(2,6-difluoro-3-hydroxy-phenyl)-5H-pyrimido[1,2-a][1,4]benzodiazepin-3-one	KI	0.85 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
[7-chloro-6-(2-fluoro-5-hydroxy-phenyl)-8-(trifluoromethyl)-4H-imidazo[1,5-a][1,4]benzodiazepin-3-yl]-(3-methoxyazetidin-1-yl)methanone	KI	0.85 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
3-[7-chloro-1-methyl-8-(trifluoromethyl)-4H-[1,2,4]triazolo[4,3-a][1,4]benzodiazepin-6-yl]-4-fluoro-phenol	KI	0.86 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
4-fluoro-3-[(4S)-7-chloro-1,4-dimethyl-8-(trifluoromethyl)-4H-[1,2,4]triazolo[4,3-a][1,4]benzodiazepin-6-yl]phenol	KI	0.99 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
[7-chloro-6-(2-fluoro-5-hydroxy-phenyl)-8-(trifluoromethyl)-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepin-2-yl]-(3-methoxyazetidin-1-yl)methanone	KI	1.04 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
(5S)-8,9-dichloro-7-(2,6-difluoro-3-hydroxy-phenyl)-5-methyl-5H-pyrimido[1,2-a][1,4]benzodiazepin-3-one	KI	1.3 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
[7,8-dichloro-6-(2-fluoro-5-hydroxy-phenyl)-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepin-2-yl]-(3-methoxyazetidin-1-yl)methanone	KI	1.4 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
8-chloro-7-(2-fluoro-5-hydroxy-phenyl)-9-(trifluoromethyl)-5H-pyrimido[1,2-a][1,4]benzodiazepin-3-one	KI	1.54 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
6-[(4S)-7-chloro-2,4-dimethyl-8-(trifluoromethyl)-4H-imidazo[1,2-a][1,4]benzodiazepin-6-yl]-5-fluoro-pyridin-2-ol	KI	2.07 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
2,4-difluoro-3-[(4S)-7,8-dichloro-1,4-dimethyl-4H-[1,2,4]triazolo[4,3-a][1,4]benzodiazepin-6-yl]phenol	KI	2.12 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
6-[(4S)-7-chloro-2,4-dimethyl-8-(trifluoromethyl)-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepin-6-yl]-5-fluoro-pyridin-2-ol	KI	2.32 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
5-chloro-6-[(4S)-7-chloro-2,4-dimethyl-8-(trifluoromethyl)-4H-imidazo[1,2-a][1,4]benzodiazepin-6-yl]pyridin-2-ol	KI	2.8 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
6-[(4S)-7-chloro-1,4-dimethyl-8-(trifluoromethyl)-4H-imidazo[1,2-a][1,4]benzodiazepin-6-yl]-5-fluoro-pyridin-2-ol	KI	3.08 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
5-chloro-6-[(4S)-7-chloro-1,4-dimethyl-8-(trifluoromethyl)-4H-imidazo[1,2-a][1,4]benzodiazepin-6-yl]pyridin-2-ol	KI	3.25 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
6-[[3-(5-chloro-2-pyridinyl)-5-methyl-1,2-oxazol-4-yl]methoxy]-N-(oxan-4-yl)pyridazine-3-carboxamide	KI	3.4 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
5-chloro-6-[(4S)-7-chloro-2,4-dimethyl-8-(trifluoromethyl)-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepin-6-yl]pyridin-2-ol	KI	3.63 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
(4S)-7-chloro-N-cyclopropyl-6-(3-fluoro-6-hydroxy-2-pyridyl)-4-methyl-8-(trifluoromethyl)-4H-imidazo[1,2-a][1,4]benzodiazepine-2-carboxamide	KI	4.4 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
(4S)-7-chloro-6-(3-fluoro-6-hydroxy-2-pyridyl)-N-isopropyl-4-methyl-8-(trifluoromethyl)-4H-imidazo[1,2-a][1,4]benzodiazepine-2-carboxamide	KI	4.7 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
6-[(4S)-7-chloro-1,4-dimethyl-8-(trifluoromethyl)-4H-[1,2,4]triazolo[4,3-a][1,4]benzodiazepin-6-yl]-5-fluoro-pyridin-2-ol	KI	5.02 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
[(4S)-7-chloro-6-(3-fluoro-6-hydroxy-2-pyridyl)-4-methyl-8-(trifluoromethyl)-4H-[1,2,4]triazolo[1,5-a][1,4]benzo diazepin-2-yl]-(3-fluoroazetidin-1-yl)methanone	KI	5.51 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
[(4S)-7,8-dichloro-6-(2,6-difluorophenyl)-4-methyl-3a,4-dihydro-1H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepin-2-yl]-(1,1-dioxo-1,4-thiazinan-4-yl)methanone	KI	8.25 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
(4S)-7,8-dichloro-6-(2,6-difluorophenyl)-2,4-dimethyl-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepine	KI	8.3 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
azetidin-1-yl-[(4S)-7,8-dichloro-6-(2,6-difluorophenyl)-4-methyl-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepin-2-yl]methanone	KI	8.5 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
6-[(10S)-6-chloro-10-methyl-5-(trifluoromethyl)-1,9,12-triazatetracyclo[9.6.0.02,7.013,17]heptadeca-2(7),3,5,8,11,13	KI	8.6 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
azetidin-1-yl-[(4S)-7-chloro-6-(3-fluoro-6-hydroxy-2-pyridyl)-4-methyl-8-(trifluoromethyl)-4H-imidazo[1,2-a][1,4]benzodiazepin-2-yl]methanone	KI	8.77 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
[(4S)-7,8-dichloro-6-(2,6-difluorophenyl)-4-methyl-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepin-2-yl]-(3-methoxyazetidin-1-yl)methanone	KI	9.2 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
(4S)-7,8-dichloro-6-(2,6-difluorophenyl)-N-(2-hydroxyethyl)-4-methyl-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepine-2-carboxamide	KI	15.2 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
5-chloro-6-[(4S)-7-chloro-1,4-dimethyl-8-(trifluoromethyl)-4H-[1,2,4]triazolo[4,3-a][1,4]benzodiazepin-6-yl]pyridin-2-ol	KI	20.9 nM	US-12344614: Benzodiazepine derivatives as GABA a GAMMA1 PAM
[(4S)-7-chloro-6-(2,6-difluorophenyl)-4-methyl-8-(trifluoromethyl)-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepin-2-yl]-(1,1-dioxo-1,4-thiazinan-4-yl)methanone	KI	23.2 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
(3-hydroxyazetidin-1-yl)-[(4S)-7-chloro-6-(2,6-difluorophenyl)-4-methyl-8-(trifluoromethyl)-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepin-2-yl]methanone	KI	23.9 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
[(4S)-7-chloro-6-(2,6-difluorophenyl)-4-methyl-8-(trifluoroethyl)-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepin-2-yl]-(3-methoxyazetidin-1-yl)methanone	KI	29 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
(7,8-dichloro-6-(2-fluorophenyl)-4H-benzo[f]imidazo[1,2-a][1,4]diazepin-2-yl)(3-methoxyazetidin-1-yl)methanone	KI	35.1 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
6-[(5-methyl-3-pyridin-3-yl-1,2-oxazol-4-yl)methoxy]-N-(oxan-4-yl)pyridazine-3-carboxamide	KI	35.6 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
azetidin-1-yl-[(4S)-7-chloro-6-(2,6-difluorophenyl)-4-methyl-8-(trifluoromethyl)-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepin-2-yl]methanone	KI	38.1 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
3-[(4S)-7-chloro-6-(2,6-difluorophenyl)-4-methyl-8-(trifluoromethyl)-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepin-2-yl]oxazolidin-2-one	KI	39.1 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
(4S)-7-chloro-6-(2,6-difluorophenyl)-N-[(2S)-2-hydroxypropyl]-4-methyl-8-(trifluoromethyl)-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepine-2-carboxamide	KI	40.8 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
1-[(4S)-7-chloro-6-(2,6-difluorophenyl)-4-methyl-8-(trifluoromethyl)-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepin-2-yl]-3-methyl-urea	KI	49.9 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
(4S)-7-chloro-6-(2,6-difluorophenyl)-N-(2-hydroxyethyl)-4-methyl-8-(trifluoromethyl)-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepine-2-carboxamide	KI	65.1 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
(4S)-7-chloro-6-(3-fluoro-2-pyridyl)-N-(2-hydroxy-2-methyl-propyl)-4-methyl-8-(trifluoromethyl)-4H-imidazo[1,2-a][1,4]benzodiazepine-2-carboxamide	KI	70.4 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
(4S)-8-bromo-7-chloro-6-(2,6-difluorophenyl)-1,4-dimethyl-4H-imidazo[1,2-a][1,4]benzodiazepine	KI	94.5 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM
(4S)-7-chloro-6-(2,6-difluorophenyl)-N’,N’,4-trimethyl-8-(trifluromethyl)-4H-[1,2,4]triazolo[1,5-a][1,4]benzodiazepine-2-carbohydrazide	KI	99.2 nM	US-12365687: Benzodiazepine derivatives as GABA A gamma 1 PAM

ChEMBL bioactivities

474 potent at pChembl≥5 of 511 total, top 50 by pChembl (potency: 10 = 0.1 nM, 6 = 1 µM).

pChembl	Type	Value	Unit	Molecule
10.00	IC50	0.1	nM	CHEMBL454349
9.80	IC50	0.16	nM	CHEMBL309517
9.70	IC50	0.2	nM	CHEMBL4747460
9.62	IC50	0.24	nM	CHEMBL79037
9.40	IC50	0.3981	nM	CGS-8216
9.40	IC50	0.4	nM	CHEMBL1271047
9.34	IC50	0.46	nM	CHEMBL78730
9.31	IC50	0.49	nM	CHEMBL76263
9.30	IC50	0.5	nM	CHEMBL5266498
9.30	IC50	0.5	nM	CHEMBL5290464
9.30	IC50	0.5	nM	CHEMBL5280240
9.30	IC50	0.5	nM	CHEMBL49141
9.30	IC50	0.5	nM	CHEMBL1518572
9.22	IC50	0.6026	nM	CGS-9896
9.22	IC50	0.6	nM	CHEMBL419096
9.12	IC50	0.76	nM	CHEMBL540583
9.10	IC50	0.79	nM	CHEMBL77226
9.10	IC50	0.8	nM	CHEMBL5265845
9.10	IC50	0.8	nM	CHEMBL5291368
9.10	IC50	0.8	nM	CHEMBL5271392
9.07	Ki	0.85	nM	CLONAZEPAM
9.05	Ki	0.9	nM	FLUMAZENIL
9.05	IC50	0.9	nM	CHEMBL5285377
9.00	Ki	1	nM	CHEMBL3410222
9.00	IC50	1	nM	BETA-CCM
9.00	IC50	1	nM	CHEMBL454606
9.00	IC50	1	nM	CHEMBL1337028
9.00	IC50	1	nM	CHEMBL509197
9.00	IC50	1	nM	ABECARNIL
9.00	Ki	1	nM	CHEMBL54341
9.00	Ki	1	nM	CHEMBL348367
8.96	IC50	1.1	nM	CHEMBL444586
8.77	Ki	1.7	nM	CHEMBL154342
8.74	IC50	1.8	nM	CHEMBL444050
8.70	IC50	2	nM	MUSCIMOL
8.66	IC50	2.2	nM	CHEMBL80610
8.64	IC50	2.3	nM	FIPRONIL
8.55	IC50	2.8	nM	CHEMBL75642
8.55	Ki	2.8	nM	CHEMBL372281
8.55	IC50	2.8	nM	DELORAZEPAM
8.52	Ki	3	nM	CHEMBL265547
8.52	IC50	3	nM	CHEMBL510764
8.52	IC50	3	nM	CHEMBL5266558
8.52	IC50	3	nM	CHEMBL2262044
8.52	IC50	3	nM	CHEMBL499814
8.52	IC50	3	nM	CHEMBL1161036
8.48	IC50	3.3	nM	CHEMBL75642
8.40	IC50	3.981	nM	CHEMBL301605
8.40	Ki	4	nM	CHEMBL330116
8.40	Ki	4	nM	CHEMBL3407528

PubChem BioAssay actives

409 with measured affinity, of 878 total; 50 most potent distinct compounds. Largely complementary to BindingDB; screening values are coarse (µM, 4 dp), so sub-nM hits tie at the floor.

Compound	Assay	Type	Value	Unit
2-(4-methoxyphenyl)-1H-pyrazolo[4,3-c]quinolin-3-one	40988: Inhibition on Benzodiazepine receptor	ic50	0.0001	uM
ethyl 4-(methoxymethyl)-5-phenylmethoxy-9H-pyrido[3,4-b]indole-3-carboxylate	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0002	uM
ethyl 4-methyl-5-propan-2-yloxy-9H-pyrido[3,4-b]indole-3-carboxylate	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0004	uM
2-phenyl-3aH-pyrazolo[4,3-c]quinolin-3-one	40988: Inhibition on Benzodiazepine receptor	ic50	0.0004	uM
ethyl 4-(methoxymethyl)-6-phenylmethoxy-9H-pyrido[3,4-b]indole-3-carboxylate	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0005	uM
ethyl 4-(methoxymethyl)-9H-pyrido[3,4-b]indole-3-carboxylate	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0005	uM
ethyl 6-methoxy-4-(methoxymethyl)-9H-pyrido[3,4-b]indole-3-carboxylate	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0005	uM
ethyl 4-(methoxymethyl)-6-propoxy-9H-pyrido[3,4-b]indole-3-carboxylate	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0005	uM
ethyl 6-hydroxy-4-(methoxymethyl)-9H-pyrido[3,4-b]indole-3-carboxylate	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0005	uM
2-(4-chlorophenyl)-3aH-pyrazolo[4,3-c]quinolin-3-one	40988: Inhibition on Benzodiazepine receptor	ic50	0.0006	uM
5-[[3-(1,3-benzodioxol-5-yl)-6-iminopyridazin-1-yl]methyl]-1,2-thiazol-3-one;hydrobromide	72153: Affinity for gamma-aminobutyric-acid A receptor measured by its ability to displace [3H]gabazine antagonist from rat brain preparations.	ic50	0.0008	uM
propan-2-yl 16-(methoxymethyl)-3,6,11,14-tetrazatetracyclo[8.7.0.02,7.012,17]heptadeca-1(10),2,4,6,8,12,14,16-octaene-15-carboxylate	1932305: Inhibition of GABAA receptor (unknown origin)	ic50	0.0008	uM
propan-2-yl 4-(methoxymethyl)-6,9,14,21-tetrazapentacyclo[11.8.0.02,10.03,8.015,20]henicosa-1(21),2,4,6,8,10,12,15(20)-octaene-5-carboxylate	1932305: Inhibition of GABAA receptor (unknown origin)	ic50	0.0008	uM
propan-2-yl 15-(methoxymethyl)-4-propan-2-yl-5-oxa-10,13-diazatetracyclo[7.7.0.02,6.011,16]hexadeca-1(9),2(6),3,7,11,13,15-heptaene-14-carboxylate	1932305: Inhibition of GABAA receptor (unknown origin)	ic50	0.0008	uM
Clonazepam	239299: Displacement of [3H]flumazenil from bovine benzodiazepine receptor GABA-A channel of brain membranes	ki	0.0008	uM
15-(methoxymethyl)-14-(5-methyl-1,2-oxazol-3-yl)-4-propan-2-yl-5-oxa-3,10,13-triazatetracyclo[7.7.0.02,6.011,16]hexadeca-1(9),2(6),3,7,11,13,15-heptaene	1932305: Inhibition of GABAA receptor (unknown origin)	ic50	0.0009	uM
Flumazenil	239299: Displacement of [3H]flumazenil from bovine benzodiazepine receptor GABA-A channel of brain membranes	ki	0.0009	uM
ethyl 9H-pyrido[3,4-b]indole-3-carboxylate	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0010	uM
methyl 9H-pyrido[3,4-b]indole-3-carboxylate	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0010	uM
propyl 9H-pyrido[3,4-b]indole-3-carboxylate	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0010	uM
tert-butyl 9H-pyrido[3,4-b]indole-3-carboxylate	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0010	uM
7-chloro-5-methyl-3-(2-phenylethynyl)-4H-imidazo[1,5-a][1,4]benzodiazepin-6-one	1196597: Displacement of [3H]flumazenil from rat cortex GABAA receptor BDZ binding site expressed in HEK293 cells by competition binding assay	ki	0.0010	uM
6-bromo-2-(3-nitrophenyl)chromen-4-one	72729: Binding affinity towards benzodiazepine site in GABAA receptor	ki	0.0010	uM
(2-methoxyphenyl)methyl 8-chloro-5-oxido-3,3a-dihydropyrazolo[5,1-c][1,2,4]benzotriazin-5-ium-3-carboxylate	41860: Binding affinity towards Benzodiazepine receptor from bovine brain membrane using [3H]Ro-151788 as radioligand	ki	0.0010	uM
propan-2-yl 4-(methoxymethyl)-6-phenylmethoxy-9H-pyrido[3,4-b]indole-3-carboxylate	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0010	uM
(2-methoxyphenyl)methyl 8-chloropyrazolo[5,1-c][1,2,4]benzotriazine-3-carboxylate	41860: Binding affinity towards Benzodiazepine receptor from bovine brain membrane using [3H]Ro-151788 as radioligand	ki	0.0017	uM
5-(aminomethyl)-1,2-oxazol-3-one	72155: Binding affinity in vivo for gamma-aminobutyric-acid A receptor measured by its ability to displace [3H]-GABA agonist from rat brain preparations after iv injection.	ic50	0.0020	uM
Fipronil	242615: In vitro insecticidal activity as inhibition of [3H]EBOB binding to Gamma-aminobutyric acid GABA-A receptor of housefly neuronal membranes	ic50	0.0023	uM
9-chloro-2-phenyl-6H-triazolo[1,2-a][1,2,4]benzotriazine-1,5-dione	239299: Displacement of [3H]flumazenil from bovine benzodiazepine receptor GABA-A channel of brain membranes	ki	0.0028	uM
7-chloro-5-(2-chlorophenyl)-1,3-dihydro-1,4-benzodiazepin-2-one	40974: In vitro displacement of [3H]-diazepam from GABA-A Benzodiazepine receptor	ic50	0.0028	uM
3-isothiocyanato-9H-pyrido[3,4-b]indole	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0030	uM
9H-pyrido[3,4-b]indole-3-carbonitrile	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0030	uM
3-(6,7-difluoro-1,3-benzoxazol-2-yl)-1,3a,4,5,6,7,8,8a-octahydrocyclohepta[d]imidazol-2-one	40986: Binding affinity against GABA-A benzodiazepine receptor	ki	0.0030	uM
(5aR,7R,9aS,11aS)-2-amino-7-hydroxy-9a,11a-dimethyl-3,3a,3b,4,5,5a,6,7,8,9,9b,11-dodecahydronaphtho[2,1-e]indol-10-one	72153: Affinity for gamma-aminobutyric-acid A receptor measured by its ability to displace [3H]gabazine antagonist from rat brain preparations.	ic50	0.0030	uM
3-propoxy-9H-pyrido[3,4-b]indole	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0030	uM
1-(9H-pyrido[3,4-b]indol-3-yl)butan-1-one	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0030	uM
8-fluoro-5-methyl-3-(2-phenylethynyl)-4H-imidazo[1,5-a][1,4]benzodiazepin-6-one	1196597: Displacement of [3H]flumazenil from rat cortex GABAA receptor BDZ binding site expressed in HEK293 cells by competition binding assay	ki	0.0040	uM
methyl 6-hydroxy-9H-pyrido[3,4-b]indole-3-carboxylate	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0040	uM
2-phenyl-6H-[1,2,4]triazolo[1,5-c]quinazolin-5-one	40986: Binding affinity against GABA-A benzodiazepine receptor	ki	0.0040	uM
17-chloro-9,12,20-triazapentacyclo[11.7.0.02,10.03,8.014,19]icosa-1(13),2(10),3,5,7,11,14,16,18-nonaene	1932305: Inhibition of GABAA receptor (unknown origin)	ic50	0.0040	uM
5-nitro-9,12,20-triazapentacyclo[11.7.0.02,10.03,8.014,19]icosa-1(13),2(10),3,5,7,11,14,16,18-nonaene	1932305: Inhibition of GABAA receptor (unknown origin)	ic50	0.0040	uM
5-bromo-9,12,20-triazapentacyclo[11.7.0.02,10.03,8.014,19]icosa-1(13),2(10),3,5,7,11,14,16,18-nonaene	1932305: Inhibition of GABAA receptor (unknown origin)	ic50	0.0040	uM
17-methoxy-9,12,20-triazapentacyclo[11.7.0.02,10.03,8.014,19]icosa-1(13),2(10),3,5,7,11,14,16,18-nonaene	1932305: Inhibition of GABAA receptor (unknown origin)	ic50	0.0040	uM
9,12,20-triazapentacyclo[11.7.0.02,10.03,8.014,19]icosa-1(13),2(10),3,5,7,11,14,16,18-nonaene	1932305: Inhibition of GABAA receptor (unknown origin)	ic50	0.0040	uM
methyl 6-(benzylamino)-9H-pyrido[3,4-b]indole-3-carboxylate	1932290: Displacement of [3H]flunitrazepam from GABAA (unknown origin ) receptor by radioligand binding assay	ic50	0.0040	uM
Diazepam	1934094: Inhibition of GABA-A receptor (unknown origin)	ic50	0.0042	uM
4-acetyl-13-(4-chlorophenyl)-5-thia-13,14-diazatricyclo[8.4.0.02,6]tetradeca-1(10),2(6),3-trien-12-one	40977: Binding affinity for GABA-A Benzodiazepine receptor	ki	0.0042	uM
1-[2,6-dichloro-4-(trifluoromethyl)phenyl]-4-(trifluoromethylsulfinyl)pyrazole	242615: In vitro insecticidal activity as inhibition of [3H]EBOB binding to Gamma-aminobutyric acid GABA-A receptor of housefly neuronal membranes	ic50	0.0042	uM
2-amino-N-[4-chloro-2-(2-chlorobenzoyl)phenyl]acetamide	40974: In vitro displacement of [3H]-diazepam from GABA-A Benzodiazepine receptor	ic50	0.0048	uM
3-(7-fluoro-1,3-benzoxazol-2-yl)-1,3a,4,5,6,7,8,8a-octahydrocyclohepta[d]imidazol-2-one	40986: Binding affinity against GABA-A benzodiazepine receptor	ki	0.0050	uM

CTD chemical–gene interactions

10 total (human), top 10 by PubMed support.

Chemical	Actions (top 5)	PubMed papers
CGP 52608	affects binding, increases reaction	1
Resveratrol	decreases expression, affects cotreatment	1
Air Pollutants	affects methylation, increases abundance	1
Benzo(a)pyrene	decreases methylation	1
Copper	affects cotreatment, decreases expression	1
gamma-Aminobutyric Acid	increases activity, affects binding	1
Valproic Acid	decreases methylation	1
8-Bromo Cyclic Adenosine Monophosphate	increases expression	1
Aflatoxin B1	decreases methylation	1
Particulate Matter	increases abundance, affects methylation	1

ChEMBL screening assays

188 unique, capped per target: 169 binding, 15 functional, 3 toxicity, 1 admet

Representative assays (with source publication via chembl_document):

Assay ID	Type	Description	Source paper
CHEMBL3363914	Binding	Displacement of [3H]Muscimol from rat GABAA receptor at 10 uM after 90 mins by microbeta counting analysis	Griseorhodins D-F, neuroactive intermediates and end products of post-PKS tailoring modification in Griseorhodin biosynthesis. — J Nat Prod
CHEMBL4810229	ADMET	Inhibition of GABA A receptor (unknown origin) at 0.1 to 1 uM	Discovery of Pemigatinib: A Potent and Selective Fibroblast Growth Factor Receptor (FGFR) Inhibitor. — J Med Chem
CHEMBL5335653	Toxicity	Antagonist activity at GABA-A (unknown origin)	Discovery of a Novel Bifunctional Steroid Analog, YXG-158, as an Androgen Receptor Degrader and CYP17A1 Inhibitor for the Treatment of Enzalutamide-Resistant Prostate Cancer. — J Med Chem

Cellosaurus cell lines

1 cell lines: 1 transformed cell line

First 10 cell lines (id-ordered, not curated):

Cellosaurus	Name	Category	Sex
CVCL_YA48	IDG-HEK293T-GABRG1-V5-OE	Transformed cell line	Female

Clinical trials (associated diseases)

312 trials via MONDO — disease-level, not drug-specific.

Trial	Phase	Status	Title
NCT00029224	PHASE4	COMPLETED	Treatment With Zoledronic Acid in Patients With Breast Cancer, Multiple Myeloma, and Prostate Cancer With Cancer Related Bone Lesions
NCT00035997	PHASE4	COMPLETED	Open-label Trial on the Effect of I.V. Zoledronic Acid 4 mg on Bone Density in Hormone Sensitive Prostate Cancer Patients With Bone Metastasis
NCT00063609	PHASE4	COMPLETED	The Effect of Zoledronic Acid on Bone Loss in Prostate Cancer Patients Undergoing Androgen Deprivation Therapy
NCT00103623	PHASE4	SUSPENDED	The Plenaxis® Experience Study
NCT00106392	PHASE4	COMPLETED	A Safety and Efficacy Study of Prograf in the Prevention of Erectile Dysfunction After Radical Prostatectomy
NCT00185029	PHASE4	UNKNOWN	MR-Lymphography and Lymph Node Staging in Prostate Cancer
NCT00199485	PHASE4	COMPLETED	Angelica Sinensis for the Treatment of Hot Flashes in Men Undergoing LHRH Therapy for Prostate Cancer
NCT00219219	PHASE4	COMPLETED	Zoledronic Acid in the Prevention of Skeletal-related Events in Hormone Refractory and Hormone-sensitive Prostate Cancer Patients With Bone Metastases
NCT00219271	PHASE4	COMPLETED	Effect Of Zoledronic Acid On Circulating And Bone Marrow-Residing Prostate Cancer Cells In Patients With Clinically Localized Prostate Cancer
NCT00237146	PHASE4	COMPLETED	Study to Evaluate Zoledronic Acid on Quality of Life and Skeletal-related Events as Adjuvant Treatment in Patients With Hormone-naïve Prostate Cancer and Bone Metastasis Who Have Undergone Orchiectomy
NCT00242554	PHASE4	COMPLETED	Open-label Phase IV Clinical Trial to Evaluate the Safety and Tolerability of Zoledronic Acid in Patients With Prostate Cancer and Bone Metastases
NCT00280098	PHASE4	COMPLETED	Docetaxel in the Treatment of Hormone Refractory Prostate Cancer
NCT00293696	PHASE4	COMPLETED	Casodex/Zoladex Biomarkers in Localised Prostate Cancer
NCT00334139	PHASE4	COMPLETED	Effect of Zoledronic Acid on Bone Metabolism in Patients With Bone Metastasis and Prostate or Breast Cancer
NCT00375765	PHASE4	COMPLETED	Effects On Dihydrotestosterone Regulated Gene Expression In Benign Prostatic Hyperplasia Or Prostate Cancer
NCT00391690	PHASE4	COMPLETED	Evaluation of Bone Markers as Diagnostic Tools for Early Detection of Bone Metastases in Patients With High Risk Prostate Cancer
NCT00422708	PHASE4	COMPLETED	Local Anesthesia for Prostate Biopsy
NCT00526331	PHASE4	COMPLETED	Evaluation of Arterial Pressure Based Cardiac Output for Goal-Directed Perioperative Therapy
NCT00590213	PHASE4	COMPLETED	Compare the Value of Prophylactic Versus Therapeutic Breast Radiotherapy in CASODEX
NCT00629330	PHASE4	TERMINATED	Dissemination of Prostate Cancer Screening to PCP’s in African American Communities
NCT00771966	PHASE4	COMPLETED	Radical Prostatectomy and Perioperative Fluid Therapy
NCT00805701	PHASE4	COMPLETED	Study Assessing The Efficacy And Safety Of Avodart (Dutasteride) At Improving Urinary Symptoms In Men With Prostate Cancer Who Are Undergoing Seed Implantation
NCT00859027	PHASE4	COMPLETED	Effect Of Risedronate On Bone Mass In Older Men Receiving Neoadjuvant Therapy For Prostate Cancer
NCT00906269	PHASE4	UNKNOWN	Can Hyperbaric Oxygen Improve Erectile Function Following Surgery for Prostate Cancer
NCT00953277	PHASE4	COMPLETED	Study of Nerve Reconstruction Using AVANCE in Subjects Who Undergo Robotic Assisted Prostatectomy for Treatment of Prostate Cancer
NCT00982800	PHASE4	COMPLETED	Does Postoperative Gabapentin Reduce Pain, Opioid Consumption and Anxiety and Have a Positive Effect on Health Related Quality of Life After Radical Prostatectomy?
NCT01083199	PHASE4	COMPLETED	Global Performance Evaluation of the AMS CONTINUUM™ Device
NCT01136226	PHASE4	COMPLETED	Evaluate Recovery of Testosterone for Patients Using Eligard
NCT01161563	PHASE4	COMPLETED	Randomized Crossover Trial to Assess the Tolerability of Gonadotropin Releasing Hormone (GnRH) Analogue Administration
NCT01230905	PHASE4	COMPLETED	Study to Monitor the Effects of Androgen Suppression Treatment on the Heart
NCT01296672	PHASE4	COMPLETED	3 Month Finasteride Challenge Test Can Significantly Improve the Performance of Screening for Prostate Cancer
NCT01365143	PHASE4	TERMINATED	Prospective Randomized Trial Comparing Robotic Versus Open Radical Prostatectomy
NCT01379742	PHASE4	UNKNOWN	Comparison of Between ThinSeed™ and OncoSeed™ for Permanent Prostate Brachytherapy
NCT01486563	PHASE4	COMPLETED	Hydroxyethyl Starch and Renal Function After Radical Prostatectomy
NCT01511874	PHASE4	COMPLETED	Efficacy and Safety Study of ELIGARD 22.5mg With Prostate Cancer
NCT01512472	PHASE4	TERMINATED	Firmagon (Degarelix) Intermittent Therapy
NCT01547416	PHASE4	COMPLETED	The Effect of Combined General/Epidural Anesthesia Versus General Anesthesia on Diaphragmatic Function
NCT01571544	PHASE4	COMPLETED	The Use of Thermal Suits as Preventing Hypothermia During Surgery
NCT01581749	PHASE4	UNKNOWN	Evaluation of Truebeam for Low-Intermediate Risk Prostate Cancer
NCT01649635	PHASE4	COMPLETED	Study of Cabazitaxel Combined With Prednisone and Prophylaxis of Neutropenia Complications in the Treatment of Patients With Metastatic Castration-resistant Prostate Cancer