HNRNPA0

Summary

HNRNPA0 (heterogeneous nuclear ribonucleoprotein A0, HGNC:5030) is a protein-coding gene on chromosome 5q31.2, encoding Heterogeneous nuclear ribonucleoprotein A0 (Q13151). mRNA-binding component of ribonucleosomes.

This gene belongs to the A/B subfamily of ubiquitously expressed heterogeneous nuclear ribonucleoproteins (hnRNPs). The hnRNPs are RNA binding proteins and they complex with heterogeneous nuclear RNA (hnRNA). These proteins are associated with pre-mRNAs in the nucleus and appear to influence pre-mRNA processing and other aspects of mRNA metabolism and transport. While all of the hnRNPs are present in the nucleus, some seem to shuttle between the nucleus and the cytoplasm. The hnRNP proteins have distinct nucleic acid binding properties. The protein encoded by this gene has two repeats of quasi-RRM domains that bind RNAs, followed by a glycine-rich C-terminus.

Source: NCBI Gene 10949 — RefSeq curated summary.

At a glance

• Gene–disease (curated): Tourette syndrome (Limited, GenCC)
• Clinical variants (ClinVar): 20 total
• Druggable target: yes
• MANE Select transcript: NM_006805

Identifiers

Gene identifiers

Field	Value
HGNC ID	HGNC:5030
Approved symbol	HNRNPA0
Name	heterogeneous nuclear ribonucleoprotein A0
Location	5q31.2
Locus type	gene with protein product
Status	Approved
Ensembl gene	ENSG00000177733
Ensembl biotype	protein_coding
OMIM	609409
Entrez	10949

Gene structure

Transcript identifiers

Ensembl transcripts: 2 — 2 protein_coding

ENST00000314940, ENST00000705031

RefSeq mRNA: 1 — MANE Select: NM_006805 NM_006805

CCDS: CCDS4193

Canonical transcript exons

ENST00000314940 — 1 exons

Exon	Start	End
ENSE00001273297	137745651	137754363

Expression profiles

Bgee: expression breadth ubiquitous, 300 present calls, max score 98.91.

FANTOM5 (CAGE): breadth ubiquitous, TPM avg 121.8379 / max 833.0626, expressed in 1827 samples.

FANTOM5 promoters (3 alternative TSS)

Promoter ID	TPM avg	Samples expressed
63650	116.8846	1827
63648	3.6579	1408
63649	1.2953	508

Top tissues by expression

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

Tissue	Anatomy ID	Expression score	Quality
adult organism	UBERON:0007023	98.91	gold quality
germinal epithelium of ovary	UBERON:0001304	98.70	gold quality
endothelial cell	CL:0000115	98.15	gold quality
middle temporal gyrus	UBERON:0002771	98.10	gold quality
embryo	UBERON:0000922	98.08	gold quality
cauda epididymis	UBERON:0004360	97.99	gold quality
pigmented layer of retina	UBERON:0001782	97.92	gold quality
retina	UBERON:0000966	97.89	gold quality
pons	UBERON:0000988	97.86	gold quality
mammary duct	UBERON:0001765	97.86	gold quality
cartilage tissue	UBERON:0002418	97.86	gold quality
caput epididymis	UBERON:0004358	97.73	gold quality
blood vessel layer	UBERON:0004797	97.73	gold quality
pericardium	UBERON:0002407	97.56	gold quality
parietal pleura	UBERON:0002400	97.55	gold quality
urethra	UBERON:0000057	97.51	gold quality
skin of hip	UBERON:0001554	97.49	gold quality
ganglionic eminence	UBERON:0004023	97.48	gold quality
epithelium of mammary gland	UBERON:0003244	97.45	gold quality
trigeminal ganglion	UBERON:0001675	97.41	gold quality
corpus epididymis	UBERON:0004359	97.37	gold quality
Brodmann (1909) area 23	UBERON:0013554	97.28	gold quality
trabecular bone tissue	UBERON:0002483	97.27	gold quality
pleura	UBERON:0000977	97.21	gold quality
dorsal root ganglion	UBERON:0000044	97.12	gold quality
heart right ventricle	UBERON:0002080	97.08	gold quality
mammalian vulva	UBERON:0000997	97.05	gold quality
substantia nigra pars compacta	UBERON:0001965	97.02	gold quality
superficial temporal artery	UBERON:0001614	97.00	gold quality
upper leg skin	UBERON:0004262	97.00	gold quality

Single-cell (SCXA)

Detected in 7 experiment(s), a significant marker in 7.

Experiment	Marker?	Max mean expression
E-HCAD-4	yes	43.22
E-CURD-112	yes	40.72
E-CURD-122	yes	40.67
E-HCAD-10	yes	38.01
E-CURD-88	yes	22.41
E-CURD-46	yes	12.77
E-ANND-3	yes	8.15

Regulation

Is transcription factor: no

miRNA regulators (miRDB)

118 targeting HNRNPA0, 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-4713-3P	100.00	65.92	505
HSA-MIR-6867-5P	100.00	82.21	3464
HSA-MIR-4673	100.00	66.64	1490
HSA-MIR-4510	100.00	66.60	2050
HSA-MIR-6127	100.00	66.76	2188
HSA-MIR-6129	100.00	66.46	2080
HSA-MIR-6130	100.00	66.69	2012
HSA-MIR-6133	100.00	66.48	2064
HSA-MIR-4747-5P	100.00	67.90	2681
HSA-MIR-5196-5P	100.00	67.98	2761
HSA-MIR-196A-1-3P	99.99	72.15	2772
HSA-MIR-186-5P	99.99	70.83	3707
HSA-MIR-4789-3P	99.99	70.75	2484
HSA-MIR-4645-5P	99.98	65.81	1284
HSA-MIR-3148	99.97	75.06	6478
HSA-MIR-3065-5P	99.97	71.56	3281
HSA-MIR-493-5P	99.96	72.47	2382
HSA-MIR-101-3P	99.94	75.03	2230
HSA-MIR-552-5P	99.93	68.56	1583
HSA-MIR-6768-5P	99.92	67.36	1942
HSA-MIR-7-1-3P	99.91	71.53	4384
HSA-MIR-7-2-3P	99.91	71.40	4394
HSA-MIR-10527-5P	99.91	72.28	3754
HSA-MIR-3529-3P	99.90	73.55	3045
HSA-MIR-3151-5P	99.86	63.83	1069
HSA-MIR-221-3P	99.86	71.56	1329
HSA-MIR-222-3P	99.86	71.35	1337
HSA-MIR-544A	99.84	68.66	1965
HSA-MIR-6764-5P	99.75	67.89	2304
HSA-MIR-6885-3P	99.75	70.36	3187

Literature-anchored findings (GeneRIF, showing 6)

• Using shotgun mass spectrometry, we found this protein differentially expressed in the dorsolateral prefrontal cortex from patients with schizophrenia. (PMID:19165527)
• Knockdown of Hnrnpa0, a del(5q) gene, alters myeloid cell fate in murine cells through regulation of AU-rich transcripts. (PMID:24532040)
• Data show that heterogeneous nuclear ribonucleoprotein A0 (hnRNPA0) drives chemo-resistance of p53-mutant tumor cells via p27Kip1/Gadd45alpha mRNAs. (PMID:26602816)
• LncRNA miR205HG hinders HNRNPA0 translation: anti-oncogenic effects in esophageal carcinoma. (PMID:34821009)
• hnRNPA0 promotes MYB expression by interacting with enhancer lncRNA MY34UE-AS in human leukemia cells. (PMID:38865811)
• The interferon-regulated host factor hnRNPA0 modulates HIV-1 production by interference with LTR activity, mRNA trafficking, and programmed ribosomal frameshifting. (PMID:38899932)

Cross-species orthologs

2 orthologs

Organism	Symbol	Gene ID
mus_musculus	Hnrnpa0	ENSMUSG00000007836
rattus_norvegicus	Hnrnpa0	ENSRNOG00000039876

Paralogs (36): DAZAP1 (ENSG00000071626), CIRBP (ENSG00000099622), RBM23 (ENSG00000100461), RBM3 (ENSG00000102317), NCL (ENSG00000115053), TIA1 (ENSG00000116001), HNRNPA2B1 (ENSG00000122566), RBM19 (ENSG00000122965), RBM39 (ENSG00000131051), MSI1 (ENSG00000135097), HNRNPA1 (ENSG00000135486), HNRNPD (ENSG00000138668), HNRNPA1L2 (ENSG00000139675), RBMX (ENSG00000147274), A1CF (ENSG00000148584), TIAL1 (ENSG00000151923), RBM46 (ENSG00000151962), HNRNPDL (ENSG00000152795), MSI2 (ENSG00000153944), RBM47 (ENSG00000163694), RBMY1F (ENSG00000169800), HNRNPA3 (ENSG00000170144), RBMXL2 (ENSG00000170748), RBM4B (ENSG00000173914), RBM4 (ENSG00000173933), RBMXL3 (ENSG00000175718), TRNAU1AP (ENSG00000180098), HNRNPAB (ENSG00000197451), RBMXL1 (ENSG00000213516), HNRNPA1L3 (ENSG00000224578), RBMY1J (ENSG00000226941), RBMY1A1 (ENSG00000234414), RBMY1E (ENSG00000242389), RBMY1B (ENSG00000242875), RBMY1D (ENSG00000244395), DND1 (ENSG00000256453)

Protein

Protein identifiers

Heterogeneous nuclear ribonucleoprotein A0 — Q13151 (reviewed: Q13151)

All UniProt accessions (1): Q13151

UniProt curated annotations — full annotation on UniProt →

Function. mRNA-binding component of ribonucleosomes. Specifically binds AU-rich element (ARE)-containing mRNAs. Involved in post-transcriptional regulation of cytokines mRNAs.

Subcellular location. Nucleus.

Post-translational modifications. Phosphorylated at Ser-84 by MAPKAPK2 in response to LPS treatment, promoting stabilization of GADD45A mRNA. Arg-291 is dimethylated, probably to asymmetric dimethylarginine.

RefSeq proteins (1): NP_006796* (*=MANE)

Domains & families (InterPro)

ID	Name	Type
IPR000504	RRM_dom	Domain
IPR012677	Nucleotide-bd_a/b_plait_sf	Homologous_superfamily
IPR034801	hnRNPA0_RRM1	Domain
IPR035979	RBD_domain_sf	Homologous_superfamily

Pfam: PF00076

UniProt features (27 total): modified residue 10, cross-link 9, compositionally biased region 3, domain 2, region of interest 2, chain 1

Structure

Experimental structures (PDB)

0 structures.

Predicted structure (AlphaFold)

Model	pLDDT	Fraction very-high
AF-Q13151-F1	73.61	0.49

Functional residue map

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

Post-translational modifications (19): 84, 133, 139, 188, 284, 291, 291, 291, 80, 96, 98, 99, 106, 154, 159, 172, 176, 1, 68

Function

Pathways and Gene Ontology

Reactome pathways

1 pathways

ID	Pathway
R-HSA-9918481	Dengue Virus-Host Interactions

MSigDB gene sets: 254 (showing top): GOBP_REGULATION_OF_MRNA_CATABOLIC_PROCESS, GOBP_INFLAMMATORY_RESPONSE, TTTGTAG_MIR520D, CHIANG_LIVER_CANCER_SUBCLASS_UNANNOTATED_DN, GOBP_3_UTR_MEDIATED_MRNA_STABILIZATION, GOBP_MACROMOLECULE_CATABOLIC_PROCESS, PRAMOONJAGO_SOX4_TARGETS_DN, NKX61_01, PAX8_B, GOBP_POST_TRANSCRIPTIONAL_REGULATION_OF_GENE_EXPRESSION, CREIGHTON_ENDOCRINE_THERAPY_RESISTANCE_1, GOBP_REGULATION_OF_CATABOLIC_PROCESS, TGCTGAY_UNKNOWN, TCF11_01, SCHAEFFER_PROSTATE_DEVELOPMENT_6HR_DN

GO Biological Process (4): mRNA processing (GO:0006397), inflammatory response (GO:0006954), response to lipopolysaccharide (GO:0032496), 3’-UTR-mediated mRNA stabilization (GO:0070935)

GO Molecular Function (6): RNA binding (GO:0003723), mRNA binding (GO:0003729), protein kinase binding (GO:0019901), mRNA 3’-UTR AU-rich region binding (GO:0035925), nucleic acid binding (GO:0003676), protein binding (GO:0005515)

GO Cellular Component (4): nucleus (GO:0005634), nucleoplasm (GO:0005654), synapse (GO:0045202), ribonucleoprotein complex (GO:1990904)

Reactome top-level categories

Rollup of top-1 pathways:

Category	Pathways
Dengue Virus Infection	1

GO top-level categories

Rollup of top GO terms by namespace:

Category	Terms
binding	2
RNA processing	1
mRNA metabolic process	1
defense response	1
response to molecule of bacterial origin	1
response to lipid	1
response to oxygen-containing compound	1
mRNA stabilization	1
nucleic acid binding	1
RNA binding	1
kinase binding	1
mRNA 3’-UTR binding	1
intracellular membrane-bounded organelle	1
nuclear lumen	1
cellular anatomical structure	1
cell junction	1
protein-containing complex	1

Protein interactions and networks

STRING

2658 interactions, top by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
HNRNPA0	HNRNPU	Q00839	891
HNRNPA0	HNRNPC	P07910	841
HNRNPA0	HNRNPUL2	Q1KMD3	706
HNRNPA0	HNRNPM	P52272	676
HNRNPA0	SRSF3	P23152	654
HNRNPA0	PCBP1	Q15365	571
HNRNPA0	RBM25	P49756	543
HNRNPA0	PTBP2	Q9UKA9	526
HNRNPA0	HNRNPK	P61978	512
HNRNPA0	HNRNPH3	P31942	497
HNRNPA0	SRRM1	Q8IYB3	492
HNRNPA0	SRP54	P13624	488
HNRNPA0	NCBP3	Q53F19	485
HNRNPA0	ELAVL1	Q15717	478
HNRNPA0	NCBP1	Q09161	472

IntAct

231 interactions, top by confidence:

A	B	Type	Score
HNRNPC	KPNA3	psi-mi:“MI:0914”(association)	0.850
YBX1	HNRNPR	psi-mi:“MI:0914”(association)	0.770
N	HNRNPR	psi-mi:“MI:0914”(association)	0.730
CFTR	ESYT2	psi-mi:“MI:2364”(proximity)	0.710
USE1	NBAS	psi-mi:“MI:0914”(association)	0.640
IGF2BP1	IGF2BP3	psi-mi:“MI:0914”(association)	0.640
SF3B1	SAP18	psi-mi:“MI:0914”(association)	0.640
HNRNPA2B1	HNRNPA0	psi-mi:“MI:0915”(physical association)	0.560
HNRNPD	HNRNPDL	psi-mi:“MI:0914”(association)	0.560
HNRNPH2	PLOD2	psi-mi:“MI:0914”(association)	0.530
MTNR1A	PGRMC1	psi-mi:“MI:0914”(association)	0.530
N	RBM47	psi-mi:“MI:0914”(association)	0.530
SYNGAP1	IGF2BP3	psi-mi:“MI:0914”(association)	0.530
TNPO1	HNRNPA0	psi-mi:“MI:0915”(physical association)	0.500
CPSF6	DDX39A	psi-mi:“MI:0914”(association)	0.480
DDX21	MED19	psi-mi:“MI:2364”(proximity)	0.480
RBM45	HNRNPDL	psi-mi:“MI:0914”(association)	0.460
HNRNPA1L2	HNRNPA0	psi-mi:“MI:0915”(physical association)	0.400
ZYX	HNRNPA0	psi-mi:“MI:0915”(physical association)	0.400
SACS	HNRNPA0	psi-mi:“MI:0915”(physical association)	0.400
Ybx1	MRPS18B	psi-mi:“MI:0915”(physical association)	0.400
MAST3	PPP6C	psi-mi:“MI:0914”(association)	0.350
PXK	YBX3	psi-mi:“MI:0914”(association)	0.350
MATR3	BCLAF3	psi-mi:“MI:0914”(association)	0.350
HNRNPA1	MATR3	psi-mi:“MI:0914”(association)	0.350
HNRNPU		psi-mi:“MI:0914”(association)	0.350
Hnrnpa3	MATR3	psi-mi:“MI:0914”(association)	0.350
Fus	DDX3X	psi-mi:“MI:0914”(association)	0.350

BioGRID (483): HNRNPA0 (Affinity Capture-MS), HNRNPA0 (Affinity Capture-MS), HNRNPA0 (Affinity Capture-MS), HNRNPA0 (Affinity Capture-MS), HNRNPA0 (Affinity Capture-MS), HNRNPA0 (Affinity Capture-MS), HNRNPA0 (Affinity Capture-MS), HNRNPA0 (Affinity Capture-MS), HNRNPA0 (Affinity Capture-MS), HNRNPA0 (Affinity Capture-MS), HNRNPA0 (Affinity Capture-MS), HNRNPA0 (Biochemical Activity), HNRNPA0 (Affinity Capture-MS), HNRNPA0 (Affinity Capture-MS), HNRNPA0 (Affinity Capture-MS)

ESM2 similar proteins: A0A0A0LLY1, A0A2R8Y4L2, A5A6H4, O89086, O93235, P04256, P09651, P09867, P10979, P17130, P21522, P27484, P49310, P49311, P49312, P51968, P51989, P51991, P51992, P60824, P60825, P60826, P98179, Q03250, Q03251, Q03878, Q05966, Q13151, Q14011, Q28521, Q28IQ9, Q2HJ60, Q32P51, Q38896, Q41188, Q43472, Q5RF83, Q61B10, Q6URK4, Q8BG05

Diamond homologs: A0A0D1C8Z4, A0A2R8Y4L2, A5A6H4, A7VJC2, D0VWM8, G5EFS2, O14979, O43347, O88569, O89086, O93235, O94432, P04256, P07909, P09405, P09651, P09867, P13383, P17130, P19338, P19682, P19683, P21522, P22626, P28644, P41891, P48809, P48810, P49312, P51968, P51989, P51990, P51991, P51992, P60824, P60825, P60826, P98179, Q02926, Q03878

SIGNOR signaling

1 interactions.

A	Effect	B	Mechanism
MAPKAPK2	“up-regulates activity”	HNRNPA0	phosphorylation

Enriched among interaction partners

Reactome pathways and GO biological processes over-represented among this gene’s 194 IntAct physical interaction partners (hypergeometric vs the genome-wide background, BH-FDR, gene-set size 15–500, ranked by fold). A functional readout of the neighbourhood — distinct from this gene’s own memberships above, and biased toward well-studied / hub proteins, so read it as themes rather than proof.

Reactome pathways:

Pathway	Partners	Fold	FDR
mRNA Polyadenylation	24	16.1×	4e-20
Processing of Capped Intron-Containing Pre-mRNA	22	13.8×	6e-17
mRNA Splicing - Major Pathway	30	12.5×	4e-22
Dengue Virus Genome Translation and Replication	5	12.1×	3e-03
mRNA Splicing - Minor Pathway	6	10.3×	2e-03
mRNA Splicing	12	10.1×	3e-07
Dengue Virus-Host Interactions	22	7.7×	1e-11
SARS-CoV-2-host interactions	8	7.3×	1e-03

GO biological processes:

GO term	Partners	Fold	FDR
positive regulation of cytoplasmic translation	7	40.8×	9e-08
U2-type prespliceosome assembly	5	18.4×	5e-04
mRNA stabilization	8	17.2×	2e-06
mRNA splicing, via spliceosome	27	14.6×	8e-21
regulation of alternative mRNA splicing, via spliceosome	10	14.4×	4e-07
autophagosome maturation	6	12.4×	6e-04
negative regulation of translation	10	11.5×	2e-06
mRNA transport	7	10.8×	3e-04

Disease & clinical

Clinical variants and AI predictions

ClinVar

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

Classification	Count (floor)
Pathogenic	0
Likely pathogenic	0
Uncertain significance	19
Likely benign	0
Benign	1

Top pathogenic / likely-pathogenic (0)

SpliceAI

174 predictions. Top by Δscore:

Variant	Effect	Δscore
5:137749664:AG:A	donor_gain	0.9900
5:137749665:G:C	donor_gain	0.9900
5:137753209:A:AC	donor_gain	0.9800
5:137753233:G:GA	donor_gain	0.9800
5:137753187:AGCCG:A	donor_gain	0.9700
5:137753212:A:AC	donor_gain	0.9700
5:137753213:C:CC	donor_gain	0.9700
5:137753537:T:TA	donor_gain	0.9700
5:137753592:TGACC:T	donor_gain	0.9200
5:137753207:CTA:C	donor_gain	0.9100
5:137753208:TAT:T	donor_gain	0.9100
5:137753231:C:CT	donor_gain	0.9100
5:137753232:T:TT	donor_gain	0.9100
5:137753296:G:C	donor_gain	0.9100
5:137753230:ACTG:A	donor_gain	0.8500
5:137751947:T:TA	donor_gain	0.8400
5:137753230:A:AC	donor_gain	0.8400
5:137753231:C:CC	donor_gain	0.8400
5:137753127:C:CA	donor_gain	0.8300
5:137753236:TCC:T	donor_gain	0.8300
5:137753592:TG:T	donor_gain	0.8200
5:137753236:T:TA	donor_gain	0.7800
5:137753262:T:TA	donor_gain	0.7800
5:137753402:C:CT	donor_gain	0.7800
5:137753362:T:TA	donor_gain	0.7700
5:137753403:C:CT	donor_gain	0.7700
5:137753237:CC:C	donor_gain	0.7600
5:137753595:C:CT	donor_gain	0.7500
5:137751906:T:A	donor_gain	0.7400
5:137753591:TTGAC:T	donor_gain	0.7400

AlphaMissense

1965 scored. Top likely-pathogenic:

Variant	Protein change	am_pathogenicity
5:137753603:G:T	A155D	1.000
5:137753630:A:G	F146S	1.000
5:137753636:A:T	V144E	1.000
5:137753638:G:C	F143L	1.000
5:137753638:G:T	F143L	1.000
5:137753639:A:C	F143C	1.000
5:137753639:A:G	F143S	1.000
5:137753640:A:G	F143L	1.000
5:137753642:C:T	G142D	1.000
5:137753644:G:C	F141L	1.000
5:137753644:G:T	F141L	1.000
5:137753645:A:G	F141S	1.000
5:137753646:A:G	F141L	1.000
5:137753648:C:T	G140E	1.000
5:137753652:G:T	R139S	1.000
5:137753714:A:G	F118S	1.000
5:137753726:A:G	L114P	1.000
5:137753759:C:T	G103E	1.000
5:137753762:A:T	V102D	1.000
5:137753764:A:C	F101L	1.000
5:137753764:A:T	F101L	1.000
5:137753765:A:C	F101C	1.000
5:137753765:A:G	F101S	1.000
5:137753766:A:C	F101V	1.000
5:137753766:A:G	F101L	1.000
5:137753766:A:T	F101I	1.000
5:137753911:G:C	F52L	1.000
5:137753911:G:T	F52L	1.000
5:137753912:A:C	F52C	1.000
5:137753912:A:G	F52S	1.000

dbSNP variants (sampled 300 via entrez): RS1000328463 (5:137755130 C>T), RS1000408900 (5:137756220 T>C), RS1000644827 (5:137745429 T>C), RS1000803343 (5:137751838 T>C), RS1000828489 (5:137751439 G>A,C), RS1000902001 (5:137751118 CAACT>C), RS1001898045 (5:137750891 A>G), RS1002033627 (5:137750632 A>C), RS1002035879 (5:137745926 G>A), RS1002101796 (5:137756136 G>A), RS1002362620 (5:137750679 G>A), RS1002395153 (5:137750446 TAGG>T), RS1002514109 (5:137754827 G>T), RS1002568465 (5:137748157 C>T), RS1002621055 (5:137747915 T>A)

Disease associations

OMIM: gene MIM:609409 | disease phenotypes:

GenCC curated gene-disease

Disease	Classification	Inheritance
Tourette syndrome	Limited	Unknown

Mondo (1): Tourette syndrome (MONDO:0007661)

Orphanet (0):

HPO phenotypes

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

GWAS associations

0 associations (top):

MeSH disease descriptors (1)

Descriptor	Name	Tree numbers
D005879	Tourette Syndrome	C10.228.140.079.898; C10.228.662.825.800; C10.574.500.850; C16.320.400.820; F03.625.992.850

Drugs & pharmacology

Drug and pharmacology data

Is drug target: yes

ChEMBL targets (1): CHEMBL6066270 (SINGLE PROTEIN)

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

ChEMBL bioactivities

4 potent at pChembl≥5 of 4 total, top 4 by pChembl (potency: 10 = 0.1 nM, 6 = 1 µM).

pChembl	Type	Value	Unit	Molecule
7.76	Kd	17.37	nM	CHEMBL5653589
7.76	ED50	17.37	nM	CHEMBL5653589
5.36	Kd	4386	nM	CHEMBL3752910
5.36	ED50	4386	nM	CHEMBL3752910

PubChem BioAssay actives

2 with measured affinity, of 4 total; 2 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
4-methyl-3-[(2-methyl-6-pyridin-3-ylpyrazolo[3,4-d]pyrimidin-4-yl)amino]-N-[3-(trifluoromethyl)phenyl]benzamide	2148523: Binding affinity to human HNRNPA0 incubated for 45 mins by Kinobead based pull down assay	kd	0.0174	uM
4-methyl-3-[(1-methyl-6-pyridin-3-ylpyrazolo[3,4-d]pyrimidin-4-yl)amino]-N-[3-(trifluoromethyl)phenyl]benzamide	2148523: Binding affinity to human HNRNPA0 incubated for 45 mins by Kinobead based pull down assay	kd	4.3859	uM

CTD chemical–gene interactions

55 total (human), top 30 by PubMed support.

Chemical	Actions (top 5)	PubMed papers
Benzo(a)pyrene	decreases expression, increases methylation, increases mutagenesis	4
sodium arsenite	decreases expression, increases expression	2
tetrabromobisphenol A	decreases expression, increases response to substance, increases expression	2
Air Pollutants	increases abundance, decreases expression	2
Valproic Acid	decreases expression	2
FR900359	increases phosphorylation	1
TAK-243	increases sumoylation	1
triphenyl phosphate	affects expression	1
propylparaben	increases expression	1
lead acetate	increases expression	1
nobiletin	decreases expression, decreases reaction	1
sodium arsenate	decreases expression, decreases reaction	1
pyrogallol 1,3-dimethyl ether	affects cotreatment, affects localization, increases expression	1
methylparaben	increases expression	1
zinc chromate	decreases expression, increases abundance	1
ochratoxin A	decreases expression	1
cupric chloride	increases expression	1
cyclic 3’,5’-uridine monophosphate	affects binding	1
chromium hexavalent ion	increases abundance, decreases expression	1
chloropicrin	decreases expression	1
perfluoro-n-nonanoic acid	decreases expression	1
4-(5-benzo(1,3)dioxol-5-yl-4-pyridin-2-yl-1H-imidazol-2-yl)benzamide	affects cotreatment, increases expression	1
erucylphospho-N,N,N-trimethylpropylammonium	decreases expression	1
bisphenol B	increases expression	1
LDN 193189	affects cotreatment, increases expression	1
3-(2-hydroxy-4-(2-methylnonan-2-yl)phenyl)cyclohexan-1-ol	decreases expression	1
bisphenol AF	increases expression	1
Sunitinib	increases expression	1
Vorinostat	decreases expression	1
Leflunomide	decreases expression	1

ChEMBL screening assays

1 unique, capped per target: 1 binding

Representative assays (with source publication via chembl_document):

Assay ID	Type	Description	Source paper
CHEMBL5651565	Binding	Binding affinity to human HNRNPA0 incubated for 45 mins by Kinobead based pull down assay	NVP-BHG712: Effects of Regioisomers on the Affinity and Selectivity toward the EPHrin Family. — ChemMedChem

Cellosaurus cell lines

1 cell lines: 1 transformed cell line

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

Cellosaurus	Name	Category	Sex
CVCL_B2YW	Abcam HEK293T HNRNPA0 KO	Transformed cell line	Female

Clinical trials (associated diseases)

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

Trial	Phase	Status	Title
NCT00152750	PHASE4	UNKNOWN	Study of Clonidine on Sleep Architecture in Children With Tourette’s Syndrome (TS) and Comorbid ADHD
NCT00226824	PHASE4	TERMINATED	Safety Study of Galantamine in Tic Disorders
NCT00241176	PHASE4	COMPLETED	Open Label Trial of Aripiprazole in Children and Adolescents With Tourette’s Disorder
NCT00370838	PHASE4	COMPLETED	Comparison of Keppra and Clonidine in the Treatment of Tics
NCT01018056	PHASE4	COMPLETED	Developing New Treatments for Tourette Syndrome: Therapeutic Trials With Modulators of Glutamatergic Neurotransmission
NCT01547000	PHASE4	COMPLETED	Guanfacine in Children With Tic Disorders
NCT03239210	PHASE4	COMPLETED	Effects of Ondansetron in Obsessive-compulsive and Tic Disorders
NCT00004376	PHASE3	COMPLETED	Phase III Randomized, Double-Blind, Placebo-Controlled Study of Guanfacine for Tourette Syndrome and Attention Deficit Hyperactivity Disorder
NCT00206323	PHASE3	COMPLETED	A Randomized, Placebo-controlled, Tourette Syndrome Study.
NCT00206336	PHASE3	COMPLETED	An Open-label Study to Determine the Efficacy and Safety of Topiramate in the Treatment of Tourette Syndrome.
NCT00478842	PHASE3	COMPLETED	Pallidal Stimulation and Gilles de la Tourette Syndrome
NCT00681863	PHASE3	TERMINATED	Open-label Extension Study of Pramipexole in the Treatment of Children and Adolescents With Tourette Syndrome
NCT01501695	PHASE3	COMPLETED	Phase III Study of 5LGr to Treat Tic Disorder
NCT03087201	PHASE3	COMPLETED	CANNAbinoids in the Treatment of TICS (CANNA-TICS)
NCT03487783	PHASE3	COMPLETED	Aripiprazole Oral Solution in the Treatment of Children and Adolescents With Tourette’s Syndrome
NCT03567291	PHASE3	TERMINATED	Evaluation of Safety and Tolerability of Long-term TEV-50717 (Deutetrabenazine) for Treatment of Tourette Syndrome in Children and Adolescents
NCT03571256	PHASE3	COMPLETED	A Study to Test if TEV-50717 is Effective in Relieving Tics Associated With Tourette Syndrome (TS)
NCT06021522	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette’s Disorder
NCT00004393	PHASE2	COMPLETED	Phase II Double Blind Placebo Controlled Trial of Risperidone in Tourette Syndrome
NCT00004652	PHASE2	COMPLETED	Phase II Pilot Controlled Study of Short Vs Longer Term Pimozide (Orap) Therapy in Tourette Syndrome
NCT00231985	PHASE2	COMPLETED	Effectiveness of Behavior Therapy and Psychosocial Therapy for the Treatment of Tourette Syndrome and Chronic Tic Disorder
NCT00311909	PHASE2	COMPLETED	Thalamic Deep Brain Stimulation for Tourette Syndrome
NCT00529308	PHASE2	COMPLETED	Transcranial Magnetic Stimulation (TMS) for Individuals With Tourette’s Syndrome
NCT00558467	PHASE2	COMPLETED	Pramipexole Pilot Phase II Study in Children and Adolescents With Tourette Disorder According to DSM-IV Criteria
NCT01043549	PHASE2	TERMINATED	Repetitive Transcranial Magnetic Stimulation of the Posterior Parietal Cortex in Patients Suffering From Gilles de la Tourette Syndrome
NCT01133353	PHASE2	WITHDRAWN	A Study of the Effectiveness and Safety of Tetrabenazine MR in Pediatric Subjects With Tourette’s Syndrome
NCT01475383	PHASE2	WITHDRAWN	Study Evaluating The Safety And Efficacy Of PF-03654746 In Adult Subjects With Tourette’s Syndrome
NCT01647269	PHASE2	COMPLETED	A Trial of Bilateral Deep Brain Stimulation to the Globus Pallidus Internum in Tourette Syndrome
NCT01904773	PHASE2	COMPLETED	Safety, Tolerability, Pharmacokinetic, and Efficacy Study of AZD5213 in Adolescents With Tourette’s Disorder
NCT02102698	PHASE2	COMPLETED	Ecopipam Treatment of Tourette’s Syndrome in Subjects 7-17 Years
NCT02217007	PHASE2	WITHDRAWN	A Trial Evaluating the Efficacy, Safety, and Pharmacokinetics of SNC-102 in Subjects With Tourette Syndrome
NCT02247206	PHASE2	COMPLETED	VoIP Delivered Behavior Therapy for Tourette Syndrome
NCT02581865	PHASE2	COMPLETED	Safety and Efficacy Study of NBI-98854 in Adults With Tourette Syndrome
NCT02619084	PHASE2	COMPLETED	Subthalamic Stimulation in Tourette’s Syndrome
NCT02679079	PHASE2	COMPLETED	Safety and Efficacy Study of NBI-98854 in Children and Adolescents With Tourette Syndrome
NCT02879578	PHASE2	COMPLETED	Safety and Tolerability Study of NBI-98854 for the Treatment of Subjects With Tourette Syndrome
NCT03066193	PHASE2	COMPLETED	Efficacy of a Therapeutic Combination of Dronabinol and PEA for Tourette Syndrome
NCT03247244	PHASE2	TERMINATED	Safety and Efficacy of Cannabis in Tourette Syndrome
NCT03325010	PHASE2	COMPLETED	Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome
NCT03444038	PHASE2	COMPLETED	Open-Label Safety and Tolerability Study of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome