CAPRIN1

Summary

CAPRIN1 (cell cycle associated protein 1, HGNC:6743) is a protein-coding gene on chromosome 11p13, encoding Caprin-1 (Q14444). mRNA-binding protein that acts as a regulator of mRNAs transport, translation and/or stability, and which is involved in neurogenesis, synaptic plasticity in neurons and cell proliferation and migration in multiple cell types.

Enables several functions, including ATP binding activity; molecular condensate scaffold activity; and signaling adaptor activity. Involved in membraneless organelle assembly; positive regulation of stress granule assembly; and regulation of gene expression. Located in cell leading edge and cytosol. Is active in intracellular membraneless organelle.

Source: NCBI Gene 4076 — RefSeq curated summary.

At a glance

• Gene–disease (curated): neurodevelopmental disorder with language impairment, autism, and attention deficit-hyperactivity disorder (Definitive, GenCC) — +2 more curated relationships
• GWAS associations: 3
• Clinical variants (ClinVar): 179 total — 17 pathogenic, 9 likely-pathogenic
• Phenotypes (HPO): 55
• Druggable target: yes
• MANE Select transcript: NM_005898

Identifiers

Gene identifiers

Field	Value
HGNC ID	HGNC:6743
Approved symbol	CAPRIN1
Name	cell cycle associated protein 1
Location	11p13
Locus type	gene with protein product
Status	Approved
Aliases	caprin-1, RNG105
Ensembl gene	ENSG00000135387
Ensembl biotype	protein_coding
OMIM	601178
Entrez	4076

Gene structure

Transcript identifiers

Ensembl transcripts: 28 — 17 protein_coding, 11 protein_coding_CDS_not_defined

ENST00000341394, ENST00000389645, ENST00000526477, ENST00000526494, ENST00000528856, ENST00000528948, ENST00000529307, ENST00000530008, ENST00000530820, ENST00000531668, ENST00000532755, ENST00000532820, ENST00000533562, ENST00000533641, ENST00000533657, ENST00000534042, ENST00000534825, ENST00000866593, ENST00000866594, ENST00000866595, ENST00000919621, ENST00000919622, ENST00000919623, ENST00000919624, ENST00000944710, ENST00000944711, ENST00000944712, ENST00000944713

RefSeq mRNA: 2 — MANE Select: NM_005898 NM_005898, NM_203364

CCDS: CCDS31453, CCDS31454

Canonical transcript exons

ENST00000341394 — 19 exons

Exon	Start	End
ENSE00000988814	34076560	34076642
ENSE00000988815	34079628	34079765
ENSE00000988816	34082825	34082877
ENSE00000988817	34082955	34083041
ENSE00000988818	34086064	34086219
ENSE00000988819	34086305	34086413
ENSE00000988820	34089395	34089456
ENSE00001186165	34051731	34051871
ENSE00001506447	34090179	34090289
ENSE00002195690	34099303	34102610
ENSE00003518991	34097698	34097761
ENSE00003582271	34071726	34071788
ENSE00003592666	34091906	34092056
ENSE00003611643	34097196	34097296
ENSE00003615455	34071901	34071987
ENSE00003622249	34090529	34090678
ENSE00003662709	34096479	34096673
ENSE00003675040	34052421	34052636
ENSE00003682460	34076236	34076474

Expression profiles

Bgee: expression breadth ubiquitous, 294 present calls, max score 99.34.

FANTOM5 (CAGE): breadth ubiquitous, TPM avg 197.4347 / max 1595.5053, expressed in 1828 samples.

FANTOM5 promoters (8 alternative TSS)

Promoter ID	TPM avg	Samples expressed
113696	63.0509	1824
113697	49.5990	1824
113698	34.0467	1819
113699	23.6887	1805
113701	16.7985	1800
113700	8.9938	1763
113703	1.2377	815
206247	0.0195	4

Top tissues by expression

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

Tissue	Anatomy ID	Expression score	Quality
cortical plate	UBERON:0005343	99.34	gold quality
ganglionic eminence	UBERON:0004023	99.25	gold quality
ventricular zone	UBERON:0003053	99.13	gold quality
embryo	UBERON:0000922	99.06	gold quality
calcaneal tendon	UBERON:0003701	98.51	gold quality
islet of Langerhans	UBERON:0000006	98.49	gold quality
endometrium	UBERON:0001295	98.40	gold quality
adrenal tissue	UBERON:0018303	98.37	gold quality
left testis	UBERON:0004533	98.31	gold quality
colonic epithelium	UBERON:0000397	98.29	gold quality
right testis	UBERON:0004534	98.20	gold quality
primordial germ cell in gonad	CL:0000670 ∩ UBERON:0000991	98.19	gold quality
stromal cell of endometrium	CL:0002255	98.19	gold quality
jejunal mucosa	UBERON:0000399	98.15	gold quality
rectum	UBERON:0001052	98.03	gold quality
secondary oocyte	CL:0000655	97.96	gold quality
adult organism	UBERON:0007023	97.91	gold quality
testis	UBERON:0000473	97.89	gold quality
ileal mucosa	UBERON:0000331	97.83	gold quality
bronchial epithelial cell	CL:0002328	97.81	gold quality
cartilage tissue	UBERON:0002418	97.78	gold quality
mucosa of sigmoid colon	UBERON:0004993	97.76	gold quality
caput epididymis	UBERON:0004358	97.71	gold quality
corpus epididymis	UBERON:0004359	97.68	gold quality
smooth muscle tissue	UBERON:0001135	97.66	gold quality
upper leg skin	UBERON:0004262	97.55	gold quality
skin of hip	UBERON:0001554	97.54	gold quality
colonic mucosa	UBERON:0000317	97.52	gold quality
gall bladder	UBERON:0002110	97.52	gold quality
tibia	UBERON:0000979	97.38	gold quality

Single-cell (SCXA)

Detected in 2 experiment(s), a significant marker in 1.

Experiment	Marker?	Max mean expression
E-ANND-3	yes	10.15
E-MTAB-6819	no	1301.71

Regulation

Is transcription factor: no

miRNA regulators (miRDB)

231 targeting CAPRIN1, 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-656-3P	100.00	72.15	2788
HSA-MIR-3613-3P	100.00	76.36	7965
HSA-MIR-4682	100.00	68.89	1258
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-98-3P	100.00	74.08	3907
HSA-MIR-1252-5P	100.00	69.80	2774
HSA-MIR-4262	100.00	73.26	3931
HSA-MIR-3925-3P	100.00	69.95	1237
HSA-MIR-4282	99.99	75.36	6408
HSA-MIR-223-3P	99.99	70.14	1140
HSA-MIR-3667-3P	99.99	67.17	1636
HSA-MIR-3662	99.99	73.82	5684
HSA-MIR-4803	99.98	71.99	3117
HSA-MIR-548P	99.98	72.25	3784
HSA-LET-7F-2-3P	99.98	70.98	2588
HSA-MIR-1185-1-3P	99.98	71.04	2593
HSA-MIR-1185-2-3P	99.98	71.04	2593
HSA-MIR-3148	99.97	75.06	6478
HSA-MIR-3688-3P	99.97	72.02	2834
HSA-MIR-607	99.97	73.62	5593
HSA-MIR-590-3P	99.96	74.34	6478
HSA-MIR-548AJ-3P	99.96	73.38	5345
HSA-MIR-548X-3P	99.96	73.38	5345
HSA-MIR-570-3P	99.96	72.41	4910
HSA-MIR-23A-3P	99.95	74.24	3163
HSA-MIR-23B-3P	99.95	74.24	3163
HSA-MIR-23C	99.95	73.92	3192
HSA-MIR-651-3P	99.94	73.48	5177

Literature-anchored findings (GeneRIF, showing 29)

• Homologous region-1 (HR-1) is a novel protein domain that has been identified near the N-terminus of human caprin-1; highly conserved in vertebrates and insects, it is also present in the human caprin-2 paralog. (PMID:14764709)
• involvement of cellular protein p137 in transcription of intermediate stage genes may regulate the transition between early and late phases of vaccinia virus replication (PMID:15471883)
• Suppression of expression of human Caprin-1 results in slowing of the proliferation rate, due to prolongation of the G1 phase of the cell cycle, formally demonstrating that Caprin-1 is essential for normal cellular proliferation. (PMID:16177067)
• Caprin-1/G3BP-1 complex is likely to regulate the transport and translation of mRNAs of proteins involved with synaptic plasticity in neurons (PMID:17210633)
• MiR-16 negatively regulate HMGA1 and caprin-1 which are involved in cell proliferation. (PMID:19250063)
• Fragile mental retardation protein interacts with the RNA-binding protein Caprin1 in neuronal RiboNucleoProtein complexes (PMID:22737234)
• These results suggest that the Japanese encephalitis virus core protein circumvents translational shutoff by inhibiting stress granule formation through an interaction with Caprin-1 and facilitates viral propagation in vitro and in vivo. (PMID:23097442)
• Cyr61/Caprin-1 co-expression was associated with worse survival. (PMID:23528710)
• G3BP1, G3BP2 and CAPRIN1 are required for translation of interferon stimulated mRNAs and are targeted by a dengue virus non-coding RNA. (PMID:24992036)
• Data show that tylophorine compounds exert anti-cancer activity predominantly by targeting and sequestering the caprin-1 protein and c-Myc mRNA associated ribonucleoprotein complex. (PMID:25669982)
• The G3BP1-Caprin1-PKR complex represents a new mode of PKR activation and is important for antiviral activity of G3BP1 and PKR during infection with mengovirus. (PMID:25784705)
• G3BP mediates the condensation of stress granules by shifting between two different states that are controlled by the phosphorylation of S149 and by binding to Caprin1 or USP10. (PMID:27022092)
• Based on insights from the structures and existing biochemical data, the existence of an evolutionarily conserved ribonucleoprotein (RNP) complex consisting of Caprin-1, FMRP and G3BP1 is proposed. (PMID:27303792)
• Data suggest that DEAD-box helicase 3 (DDX3X) physically interacts and co-localizes with poly(A)-binding cytoplasmic protein 1 (PABPC1) and caprin-1 in lamellipodia at the leading edge of spreading cells; these interactions are dependent on mRNA; depletion of DDX3X (via gene silencing with the CRISPR-Cas system) leads to decreased cell motility. These studies were conducted using MRC5 lung fibroblast cell line. (PMID:28733330)
• that high Caprin1 expression was significantly associated with worse overall survival for patients with hepatocellular carcinoma (PMID:29037839)
• this study found that different FMRP serine/threonine and CAPRIN1 tyrosine phosphorylation patterns control phase separation propensity with RNA, including subcompartmentalization, and tune deadenylation and translation rates in vitro. (PMID:31439799)
• NMR Experiments for Studies of Dilute and Condensed Protein Phases: Application to the Phase-Separating Protein CAPRIN1. (PMID:31898464)
• we knocked down three regulators respectively and found two of them (TRA2A and CAPRIN1) selectively promoted the methylations of the m6A sites co-localized with their binding targets on RNAs through physical interactions with the m6A writers. Knockdown of TRA2A increased the stabilities of the RNAs with TRA2A bound near the m6A sites and decreased the viability of cells (PMID:31912146)
• LncRNA SNHG8 induces ovarian carcinoma cells cellular process and stemness through Wnt/beta-catenin pathway. (PMID:32538821)
• Interaction hot spots for phase separation revealed by NMR studies of a CAPRIN1 condensed phase. (PMID:34074792)
• CAPRIN1 haploinsufficiency causes a neurodevelopmental disorder with language impairment, ADHD and ASD. (PMID:35979925)
• Long noncoding RNA ZNFX1-AS1 promotes the invasion and proliferation of gastric cancer cells by regulating LIN28 and CAPR1N1. (PMID:36160641)
• circCAPRIN1 interacts with STAT2 to promote tumor progression and lipid synthesis via upregulating ACC1 expression in colorectal cancer. (PMID:36328987)
• STRESS granule-associated RNA-binding protein CAPRIN1 drives cancer progression and regulates treatment response in nasopharyngeal carcinoma. (PMID:36515758)
• Caprin-1 plays a role in cell proliferation and Warburg metabolism of esophageal carcinoma by regulating METTL3 and WTAP. (PMID:36855123)
• CAPRIN1 Is Required for Control of Viral Replication Complexes by Interferon Gamma. (PMID:37052473)
• Caprin-1 binding to the critical stress granule protein G3BP1 is influenced by pH. (PMID:37161291)
• Caprin-1 influences autophagy-induced tumor growth and immune modulation in pancreatic cancer. (PMID:38082307)
• Caprin1 Bridges PRMT1 to G3BP1 and Spaces Them to Ensure Proper Stress Granule Formation. (PMID:39079611)

Cross-species orthologs

5 orthologs

Organism	Symbol	Gene ID
danio_rerio	caprin1a	ENSDARG00000009346
danio_rerio	caprin1b	ENSDARG00000054272
mus_musculus	Caprin1	ENSMUSG00000027184
rattus_norvegicus	Caprin1	ENSRNOG00000009152
drosophila_melanogaster	Capr	FBGN0042134

Paralogs (1): CAPRIN2 (ENSG00000110888)

Protein

Protein identifiers

Caprin-1 — Q14444 (reviewed: Q14444)

Alternative names: Cell cycle-associated protein 1, Cytoplasmic activation- and proliferation-associated protein 1, GPI-anchored membrane protein 1, GPI-anchored protein p137, Membrane component chromosome 11 surface marker 1, RNA granule protein 105

All UniProt accessions (3): E9PLA9, Q14444, G3V153

UniProt curated annotations — full annotation on UniProt →

Function. mRNA-binding protein that acts as a regulator of mRNAs transport, translation and/or stability, and which is involved in neurogenesis, synaptic plasticity in neurons and cell proliferation and migration in multiple cell types. Plays an essential role in cytoplasmic stress granule formation. Acts as an mRNA regulator by mediating formation of some phase-separated membraneless compartment: undergoes liquid-liquid phase separation upon binding to target mRNAs, leading to assemble mRNAs into cytoplasmic ribonucleoprotein granules that concentrate mRNAs with associated regulatory factors. Undergoes liquid-liquid phase separation following phosphorylation and interaction with FMR1, promoting formation of cytoplasmic ribonucleoprotein granules that concentrate mRNAs with factors that inhibit translation and mediate deadenylation of target mRNAs. In these cytoplasmic ribonucleoprotein granules, CAPRIN1 mediates recruitment of CNOT7 deadenylase, leading to mRNA deadenylation and degradation. Binds directly and selectively to MYC and CCND2 mRNAs. In neuronal cells, directly binds to several mRNAs associated with RNA granules, including BDNF, CAMK2A, CREB1, MAP2, NTRK2 mRNAs, as well as to GRIN1 and KPNB1 mRNAs, but not to rRNAs.

Subunit / interactions. May form homomultimers. Interacts with G3BP1; interaction is direct and promotes stress granule formation. Interacts with G3BP2; interaction is direct and promotes stress granule formation. Interacts with PQBP1. Interacts with DDX3X. Interacts (when phosphorylated by EPHA4) with FMR1; interaction with FMR1 promotes formation of a membraneless compartment. (Microbial infection) Interacts with Zika virus capsid protein C; this interaction is probably linked to the inhibition of stress granules formation by the virus. (Microbial infection) Interacts with rotavirus A non-structural protein 5; this interaction probably plays a role in the sequestration of CAPRIN1 in viral factories. (Microbial infection) Interacts with Japanese encephalitis virus capsid protein C; this interaction is involved in the suppression of the integrated stress response by the virus.

Subcellular location. Cytoplasm. Cytoplasmic ribonucleoprotein granule. Cytosol. Cell projection. Dendrite. Lamellipodium Cytoplasm.

Tissue specificity. Ubiquitous.

Post-translational modifications. Tyrosine phosphorylation by EPHA4 promotes interaction with FMR1 and liquid-liquid phase separation (LLPS) for the formation of a membraneless compartment that concentrates mRNAs with associated regulatory factors. O-glycosylated (O-GlcNAcylated), in a cell cycle-dependent manner. O-glycosylation by OGT inhibit ability to undergo liquid-liquid phase separation (LLPS).

Disease relevance. Neurodegeneration, childhood-onset, with cerebellar ataxia and cognitive decline (CONDCAC) [MIM:620636] A neurodegenerative disorder characterized by early-onset ataxia, dysarthria, cognitive decline, sensorimotor axonal neuropathy and muscle weakness. Brain imaging shows cerebellar atrophy. The disease is caused by variants affecting the gene represented in this entry. Neurodevelopmental disorder with language impairment, autism, and attention deficit-hyperactivity disorder (NEDLAAD) [MIM:620782] An autosomal dominant disorder with variable expressivity and incomplete penetrance. It is characterized by language impairment, speech delay, intellectual disability, attention deficit hyperactivity disorder and autism spectrum disorder. Additional variable features include developmental delay, seizures, skeletal anomalies, respiratory difficulties, and ophthalmologic anomalies. The disease is caused by variants affecting the gene represented in this entry.

Activity regulation. Ability to mediate liquid-liquid phase separation is regulated by ATP: moderate concentrations of ATP enhance phase separation, whereas high concentrations of ATP lead to inhibition of phase separation.

Domain organisation. The C-terminal disordered region undergoes liquid-liquid phase separation (LLPS) for the formation of a membraneless compartment that concentrates mRNAs with associated regulatory factors. CAPRIN1 molecules in the condensed phase are neutral. mRNA-binding promotes phase separation. Moderate concentrations of ATP enhance phase separation by reducing the electrostatic potential of CAPRIN1, thereby promoting intermolecular interactions. In contrast, high concentrations of ATP invert the electrostatic potential of CAPRIN1, so that CAPRIN1 molecules become negatively charged, lead to inhibition of phase separation.

Similarity. Belongs to the caprin family.

Isoforms (3)

UniProt ID	Names	Canonical?
Q14444-1	1	yes
Q14444-2	2
Q14444-3	3

RefSeq proteins (2): NP_005889, NP_976240 (=MANE)

Domains & families (InterPro)

ID	Name	Type
IPR022070	Caprin-1_C	Domain
IPR028816	Caprin	Family
IPR041637	Caprin-1_dimer	Domain

Pfam: PF12287, PF18293

UniProt features (100 total): mutagenesis site 31, modified residue 19, sequence variant 13, compositionally biased region 10, helix 8, region of interest 6, strand 3, initiator methionine 2, glycosylation site 2, splice variant 2, coiled-coil region 2, chain 1, sequence conflict 1

Structure

Experimental structures (PDB)

6 structures.

PDB	Method	Resolution (Å)
9HFU	X-RAY DIFFRACTION	1.7
6TA7	X-RAY DIFFRACTION	1.93
4WBE	X-RAY DIFFRACTION	2.05
7XHG	X-RAY DIFFRACTION	2.46
4WBP	X-RAY DIFFRACTION	2.5
8TH7	X-RAY DIFFRACTION	2.88

Predicted structure (AlphaFold)

Model	pLDDT	Fraction very-high
AF-Q14444-F1	58.64	0.29

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): 2, 10, 115, 165, 335, 343, 625, 626, 633, 636, 639, 640, 651, 662, 665, 670, 698, 698, 2

Glycosylation sites (2): 644, 649

Mutagenesis-validated functional residues (31):

Position	Phenotype
370	abolished interaction with g3bp1.
372	abolished interaction with g3bp1 and ability to promote stress granule formation.
373	abolished interaction with g3bp1.
373	increased interaction with g3bp1.
376	abolished interaction with g3bp1.
376	increased interaction with g3bp1.
377	increased interaction with g3bp1.
377	abolished interaction with g3bp1.
608	abolished ability to undergo liquid-liquid phase separation for the formation of a membraneless compartment; when associ
612	major reduction in myc and ccnd2 rna-binding; when associated with a-633 and a-690.
612	abolished ability to undergo liquid-liquid phase separation for the formation of a membraneless compartment; when associ
616	abolished ability to undergo liquid-liquid phase separation for the formation of a membraneless compartment; when associ
619	abolished ability to undergo liquid-liquid phase separation for the formation of a membraneless compartment; when associ
624–626	decreased ability to undergo liquid-liquid phase separation.
626	abolished ability to undergo liquid-liquid phase separation for the formation of a membraneless compartment; when associ
633	major reduction in myc and ccnd2 rna-binding; when associated with a-612 and a-690.
633	abolished ability to undergo liquid-liquid phase separation for the formation of a membraneless compartment; when associ
638–640	decreased ability to undergo liquid-liquid phase separation.
640	abolished ability to undergo liquid-liquid phase separation for the formation of a membraneless compartment; when associ
660–662	decreased ability to undergo liquid-liquid phase separation.
660	abolished ability to undergo liquid-liquid phase separation for the formation of a membraneless compartment; when associ
667	abolished ability to undergo liquid-liquid phase separation for the formation of a membraneless compartment; when associ
676	abolished ability to undergo liquid-liquid phase separation for the formation of a membraneless compartment; when associ
680–682	does not affect ability to undergo liquid-liquid phase separation.
684	abolished ability to undergo liquid-liquid phase separation for the formation of a membraneless compartment; when associ

Function

Pathways and Gene Ontology

Reactome pathways

0 pathways

MSigDB gene sets: 480 (showing top): GOBP_DENDRITE_DEVELOPMENT, TGGTGCT_MIR29A_MIR29B_MIR29C, RODRIGUES_THYROID_CARCINOMA_ANAPLASTIC_UP, GOBP_REGULATION_OF_CELL_MORPHOGENESIS, GOBP_REGULATION_OF_MRNA_CATABOLIC_PROCESS, GOBP_SYNAPSE_ASSEMBLY, GOBP_REGULATION_OF_DENDRITE_MORPHOGENESIS, GOBP_DENDRITIC_SPINE_DEVELOPMENT, MAZ_Q6, GRAESSMANN_APOPTOSIS_BY_DOXORUBICIN_DN, GRAESSMANN_RESPONSE_TO_MC_AND_DOXORUBICIN_DN, GOBP_MACROMOLECULE_CATABOLIC_PROCESS, TATTATA_MIR374, GOBP_NEUROGENESIS, GOBP_REGULATION_OF_NERVOUS_SYSTEM_DEVELOPMENT

GO Biological Process (15): synapse assembly (GO:0007416), intracellular mRNA localization (GO:0008298), negative regulation of translation (GO:0017148), generation of neurons (GO:0048699), positive regulation of dendrite morphogenesis (GO:0050775), positive regulation of dendritic spine morphogenesis (GO:0061003), positive regulation of stress granule assembly (GO:0062029), regulation of deadenylation-dependent decapping of nuclear-transcribed mRNA (GO:0106288), membraneless organelle assembly (GO:0140694), deadenylation-dependent decapping of nuclear-transcribed mRNA (GO:0000290), nervous system development (GO:0007399), cell differentiation (GO:0030154), stress granule assembly (GO:0034063), negative regulation of translational initiation (GO:0045947), synapse organization (GO:0050808)

GO Molecular Function (8): RNA binding (GO:0003723), mRNA binding (GO:0003729), ATP binding (GO:0005524), signaling adaptor activity (GO:0035591), molecular function activator activity (GO:0140677), molecular condensate scaffold activity (GO:0140693), nucleotide binding (GO:0000166), protein binding (GO:0005515)

GO Cellular Component (12): P-body (GO:0000932), cytoplasm (GO:0005737), cytosol (GO:0005829), cytoplasmic stress granule (GO:0010494), membrane (GO:0016020), lamellipodium (GO:0030027), dendrite (GO:0030425), cell leading edge (GO:0031252), intracellular membraneless organelle (GO:0043232), synapse (GO:0045202), cytoplasmic ribonucleoprotein granule (GO:0036464), cell projection (GO:0042995)

GO top-level categories

Rollup of top GO terms by namespace:

Category	Terms
cellular anatomical structure	5
protein-macromolecule adaptor activity	2
cytoplasmic ribonucleoprotein granule	2
intracellular anatomical structure	2
cytoplasm	2
nervous system development	1
cell junction assembly	1
synapse organization	1
RNA localization	1
translation	1
regulation of translation	1
negative regulation of gene expression	1
negative regulation of protein metabolic process	1
neurogenesis	1
positive regulation of cell morphogenesis	1
positive regulation of cell projection organization	1
dendrite morphogenesis	1
regulation of dendrite morphogenesis	1
positive regulation of neurogenesis	1
positive regulation of neuron projection development	1
positive regulation of dendrite morphogenesis	1
dendritic spine morphogenesis	1
positive regulation of dendritic spine development	1
regulation of dendritic spine morphogenesis	1
stress granule assembly	1
regulation of stress granule assembly	1
positive regulation of organelle assembly	1
deadenylation-dependent decapping of nuclear-transcribed mRNA	1
regulation of mRNA catabolic process	1
organelle assembly	1
nuclear-transcribed mRNA catabolic process, deadenylation-dependent decay	1
nuclear-transcribed mRNA catabolic process	1
mRNA methylguanosine-cap decapping	1
system development	1
cellular developmental process	1
membraneless organelle assembly	1
translational initiation	1
regulation of translational initiation	1
negative regulation of translation	1
cell junction organization	1

Protein interactions and networks

STRING

1862 interactions, top by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
CAPRIN1	G3BP1	Q13283	994
CAPRIN1	G3BP2	Q9UN86	953
CAPRIN1	NAT10	Q9H0A0	909
CAPRIN1	FMR1	Q06787	901
CAPRIN1	TIAL1	Q01085	842
CAPRIN1	TIA1	P31483	836
CAPRIN1	EIF4E	P06730	751
CAPRIN1	STAT1	P42224	740
CAPRIN1	PABPC1	P11940	724
CAPRIN1	EIF3B	P55884	721
CAPRIN1	FXR1	P51114	708
CAPRIN1	DDX6	P26196	703
CAPRIN1	OGFOD1	Q8N543	698
CAPRIN1	UBAP2L	Q14157	688
CAPRIN1	EIF4G1	Q04637	686

IntAct

184 interactions, top by confidence:

A	B	Type	Score
G3BP1	CAPRIN1	psi-mi:“MI:0914”(association)	0.840
G3BP1	CAPRIN1	psi-mi:“MI:0915”(physical association)	0.840
G3BP1	CAPRIN1	psi-mi:“MI:0403”(colocalization)	0.840
CAPRIN1	G3BP1	psi-mi:“MI:0403”(colocalization)	0.840
CAPRIN1	N	psi-mi:“MI:0915”(physical association)	0.740
G3BP2	CAPRIN1	psi-mi:“MI:0914”(association)	0.740
N	HNRNPR	psi-mi:“MI:0914”(association)	0.730
CAPRIN1	FXR2	psi-mi:“MI:0915”(physical association)	0.640
NXF1	DDX3X	psi-mi:“MI:0914”(association)	0.600
ILK	HAX1	psi-mi:“MI:0914”(association)	0.530
FMR1	ACOT7	psi-mi:“MI:0914”(association)	0.500
ESR1		psi-mi:“MI:0914”(association)	0.460
CAPRIN1	DAPK1	psi-mi:“MI:0407”(direct interaction)	0.440
CAPRIN1	MFHAS1	psi-mi:“MI:0407”(direct interaction)	0.440
FUS	DDX3X	psi-mi:“MI:0914”(association)	0.430
FUS	CAPRIN1	psi-mi:“MI:0403”(colocalization)	0.430
PES1	CAPRIN1	psi-mi:“MI:0915”(physical association)	0.400
BNIP5	CAPRIN1	psi-mi:“MI:0915”(physical association)	0.400
CAPRIN1	RBM25	psi-mi:“MI:0915”(physical association)	0.400
CAPRIN1	ABTB1	psi-mi:“MI:0915”(physical association)	0.400
Bles03		psi-mi:“MI:0915”(physical association)	0.400
	GNAT3	psi-mi:“MI:0915”(physical association)	0.400
CAPRIN1	WBP4	psi-mi:“MI:0915”(physical association)	0.400
CAPRIN1	FXR1	psi-mi:“MI:0915”(physical association)	0.370

BioGRID (604): CAPRIN1 (Affinity Capture-MS), CAPRIN1 (Affinity Capture-MS), CAPRIN1 (Affinity Capture-MS), CAPRIN1 (Affinity Capture-MS), CAPRIN1 (Affinity Capture-MS), CAPRIN1 (Affinity Capture-MS), CAPRIN1 (Affinity Capture-MS), CAPRIN1 (Affinity Capture-MS), CAPRIN1 (Co-fractionation), CAPRIN1 (Affinity Capture-MS), CAPRIN1 (Affinity Capture-MS), CAPRIN1 (Affinity Capture-MS), CAPRIN1 (Affinity Capture-MS), CAPRIN1 (Affinity Capture-MS), CAPRIN1 (Affinity Capture-MS)

ESM2 similar proteins: A0A3B3IU46, A0JMU8, A1L1K8, A2RV70, O94432, P07733, P45978, P46553, P90897, Q09801, Q09911, Q14444, Q1LZB6, Q24669, Q28F29, Q28HC9, Q2HJG4, Q5CZI8, Q5JVS0, Q5M9G3, Q5R9Q6, Q5UR41, Q5ZMS6, Q60865, Q66HC1, Q6CVS3, Q6FJC7, Q6NRP6, Q6NRY1, Q6NYG6, Q6P0F4, Q6P1U3, Q75A59, Q8CGZ0, Q8IWX8, Q8TAP9, Q8VDM6, Q91W18, Q9BTL3, Q9BUJ2

Diamond homologs: Q14444, Q1LZB6, Q5M9G3, Q5RJ80, Q60865, Q9I7D3, Q05A80, A0A060WQA3, A5PN28, B2RNN3, B2RPV6, O75973, O88992, P02746, P02747, P08125, P0C862, P23206, P23435, P25067, P25318, P31721, P63182, P83371, P86437, P98085, P98087, Q03692, Q05306, Q06577, Q13201, Q17QF9, Q4ZJM9, Q5VWW1, Q6IMN6, Q6UW01, Q7Z5L3, Q86Z23, Q8BGU2, Q8BME9

SIGNOR signaling

1 interactions.

A	Effect	B	Mechanism
CAPRIN1	“up-regulates activity”	G3BP1	binding

Enriched among interaction partners

Reactome pathways and GO biological processes over-represented among this gene’s 173 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.

GO biological processes:

GO term	Partners	Fold	FDR
stress granule assembly	6	24.1×	9e-05
negative regulation of translation	9	11.8×	9e-05
mRNA transport	6	10.5×	4e-03
cytoplasmic translation	8	9.9×	5e-04
RNA splicing	10	5.9×	2e-03
defense response to virus	10	4.6×	8e-03
positive regulation of apoptotic process	11	4.2×	8e-03

Disease & clinical

Clinical variants and AI predictions

ClinVar

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

Classification	Count (floor)
Pathogenic	17
Likely pathogenic	9
Uncertain significance	109
Likely benign	9
Benign	2

Top pathogenic / likely-pathogenic (26)

Variant ID	HGVS	Classification
1338774	NM_005898.5(CAPRIN1):c.1493_1496del (p.Ser498fs)	Pathogenic
1809826	NM_005898.5(CAPRIN1):c.977C>A (p.Ser326Ter)	Pathogenic
2580936	NM_005898.5(CAPRIN1):c.891_894del (p.Arg297fs)	Pathogenic
3069139	NM_005898.5(CAPRIN1):c.879G>A (p.Glu293=)	Pathogenic
3069140	NM_005898.5(CAPRIN1):c.892C>T (p.Gln298Ter)	Pathogenic
3069141	NM_005898.5(CAPRIN1):c.1195C>T (p.Gln399Ter)	Pathogenic
3069142	NM_005898.5(CAPRIN1):c.1744C>T (p.Gln582Ter)	Pathogenic
3342687	NM_005898.5(CAPRIN1):c.241C>T (p.Arg81Ter)	Pathogenic
3827321	NM_005898.5(CAPRIN1):c.1604del (p.Thr534_Leu535insTer)	Pathogenic
4082550	NM_005898.5(CAPRIN1):c.844G>T (p.Glu282Ter)	Pathogenic
4218956	NM_005898.5(CAPRIN1):c.215del (p.Lys72fs)	Pathogenic
4218957	NM_005898.5(CAPRIN1):c.947G>A (p.Trp316Ter)	Pathogenic
4277288	NM_005898.5(CAPRIN1):c.1689dup (p.Glu564fs)	Pathogenic
4281350	NM_005898.5(CAPRIN1):c.1635T>A (p.Tyr545Ter)	Pathogenic
4534550	NM_005898.5(CAPRIN1):c.1674_1677del (p.Thr559fs)	Pathogenic
4685117	NM_005898.5(CAPRIN1):c.1372C>T (p.Arg458Ter)	Pathogenic
4819016	NM_005898.5(CAPRIN1):c.1066A>T (p.Arg356Ter)	Pathogenic
1712044	NM_005898.5(CAPRIN1):c.1660C>T (p.His554Tyr)	Likely pathogenic
2500315	NM_005898.5(CAPRIN1):c.1654C>T (p.Gln552Ter)	Likely pathogenic
2570649	NM_005898.5(CAPRIN1):c.274C>T (p.Gln92Ter)	Likely pathogenic
3345374	NM_005898.5(CAPRIN1):c.1648del (p.Ser550fs)	Likely pathogenic
3373614	NM_005898.5(CAPRIN1):c.688+5G>A	Likely pathogenic
4278434	NM_005898.5(CAPRIN1):c.1733_1740del (p.Asp578fs)	Likely pathogenic
4294301	NM_005898.5(CAPRIN1):c.380_383del (p.Ile127fs)	Likely pathogenic
4526445	NM_005898.5(CAPRIN1):c.832G>T (p.Glu278Ter)	Likely pathogenic
982189	NM_005898.5(CAPRIN1):c.811C>T (p.Gln271Ter)	Likely pathogenic

SpliceAI

2438 predictions. Top by Δscore:

Variant	Effect	Δscore
11:34052411:C:CA	acceptor_gain	1.0000
11:34071722:ATAG:A	acceptor_gain	1.0000
11:34071723:T:G	acceptor_gain	1.0000
11:34071724:AG:A	acceptor_gain	1.0000
11:34071725:GG:G	acceptor_gain	1.0000
11:34071784:AGCTG:A	donor_loss	1.0000
11:34071785:GCTG:G	donor_gain	1.0000
11:34071786:CTGG:C	donor_loss	1.0000
11:34071787:TGGTA:T	donor_loss	1.0000
11:34071788:GG:G	donor_loss	1.0000
11:34071789:G:GA	donor_loss	1.0000
11:34071790:T:G	donor_loss	1.0000
11:34071873:GTAAA:G	acceptor_gain	1.0000
11:34071899:A:AG	acceptor_gain	1.0000
11:34071899:AG:A	acceptor_gain	1.0000
11:34071900:G:GG	acceptor_gain	1.0000
11:34071900:GG:G	acceptor_gain	1.0000
11:34071900:GGAT:G	acceptor_gain	1.0000
11:34071973:GCAC:G	donor_gain	1.0000
11:34071983:AAGAT:A	donor_gain	1.0000
11:34071984:AGAT:A	donor_gain	1.0000
11:34071984:AGATG:A	donor_loss	1.0000
11:34071985:GAT:G	donor_gain	1.0000
11:34071985:GATG:G	donor_gain	1.0000
11:34071986:AT:A	donor_gain	1.0000
11:34071987:TG:T	donor_loss	1.0000
11:34071988:G:GG	donor_gain	1.0000
11:34071988:GTAA:G	donor_loss	1.0000
11:34071989:TAA:T	donor_loss	1.0000
11:34071991:A:AG	donor_gain	1.0000

AlphaMissense

4650 scored. Top likely-pathogenic:

Variant	Protein change	am_pathogenicity
11:34052607:A:G	K63E	1.000
11:34052608:A:T	K63M	1.000
11:34052609:G:C	K63N	1.000
11:34052609:G:T	K63N	1.000
11:34052610:A:G	K64E	1.000
11:34052614:T:C	L65P	1.000
11:34052617:G:C	R66P	1.000
11:34052619:A:G	N67D	1.000
11:34052621:C:A	N67K	1.000
11:34052621:C:G	N67K	1.000
11:34052623:T:C	L68P	1.000
11:34052628:A:G	K70E	1.000
11:34052630:G:C	K70N	1.000
11:34052630:G:T	K70N	1.000
11:34052633:A:C	K71N	1.000
11:34052633:A:T	K71N	1.000
11:34071729:A:G	K74E	1.000
11:34071731:G:C	K74N	1.000
11:34071731:G:T	K74N	1.000
11:34071733:T:A	L75H	1.000
11:34071733:T:C	L75P	1.000
11:34071772:T:A	L88H	1.000
11:34071772:T:C	L88P	1.000
11:34071784:A:C	Q92P	1.000
11:34071785:G:C	Q92H	1.000
11:34071785:G:T	Q92H	1.000
11:34071787:T:C	L93P	1.000
11:34071904:G:C	A95P	1.000
11:34071905:C:A	A95D	1.000
11:34071926:T:A	V102D	1.000

dbSNP variants (sampled 300 via entrez): RS1000013866 (11:34052247 G>GCC), RS1000069736 (11:34074872 A>G), RS1000088953 (11:34052115 G>A,C,T), RS1000096011 (11:34089304 A>G), RS1000231493 (11:34057002 A>G), RS1000267971 (11:34069921 A>G), RS1000359908 (11:34061916 G>A), RS1000360840 (11:34102332 A>G,T), RS1000399329 (11:34086499 T>C,G), RS1000426706 (11:34089564 C>A,G), RS1000483389 (11:34050901 A>G), RS1000591731 (11:34101478 G>A), RS1000592358 (11:34080072 C>G,T), RS1000669241 (11:34061089 G>A,T), RS1000691442 (11:34060831 A>C,G)

Disease associations

OMIM: gene MIM:601178 | disease phenotypes: MIM:209850, MIM:254770, MIM:606904, MIM:620636, MIM:620782

GenCC curated gene-disease

Disease	Classification	Inheritance
neurodevelopmental disorder with language impairment, autism, and attention deficit-hyperactivity disorder	Definitive	Autosomal dominant
autism spectrum disorder	Limited	Autosomal dominant
neurodegeneration, childhood-onset, with cerebellar ataxia and cognitive decline	Limited	Autosomal dominant

ClinGen Gene-Disease Validity (2)

Expert-panel classifications — Definitive > Strong > Moderate > Limited > Disputed > Refuted.

Disease	Classification	Inheritance
neurodegeneration, childhood-onset, with cerebellar ataxia and cognitive decline	Moderate	AD
neurodevelopmental disorder with language impairment, autism, and attention deficit-hyperactivity disorder	Moderate	AD

Mondo (6): autism spectrum disorder (MONDO:0005258), autism (MONDO:0005260), juvenile myoclonic epilepsy (MONDO:0009696), cerebellar ataxia (MONDO:0000437), neurodegeneration, childhood-onset, with cerebellar ataxia and cognitive decline (MONDO:0957985), neurodevelopmental disorder with language impairment, autism, and attention deficit-hyperactivity disorder (MONDO:0968945)

Orphanet (4): Juvenile myoclonic epilepsy (Orphanet:307), Rare ataxia (Orphanet:102002), Moyamoya angiopathy (Orphanet:477768), NON RARE IN EUROPE: Autism (Orphanet:106)

HPO phenotypes

55 total (30 of 55 shown, HPO-id order):

HPO	Term
HP:0000006	Autosomal dominant inheritance
HP:0000201	Pierre-Robin sequence
HP:0000219	Thin upper lip vermilion
HP:0000276	Long face
HP:0000294	Low anterior hairline
HP:0000316	Hypertelorism
HP:0000347	Micrognathia
HP:0000365	Hearing impairment
HP:0000455	Broad nasal tip
HP:0000483	Astigmatism
HP:0000486	Strabismus
HP:0000490	Deeply set eye
HP:0000508	Ptosis
HP:0000540	Hypermetropia
HP:0000545	Myopia
HP:0000601	Hypotelorism
HP:0000718	Aggressive behavior
HP:0000729	Autistic behavior
HP:0000739	Anxiety
HP:0000750	Delayed speech and language development
HP:0000957	Cafe-au-lait spot
HP:0000960	Sacral dimple
HP:0001195	Single umbilical artery
HP:0001212	Prominent fingertip pads
HP:0001251	Ataxia
HP:0001260	Dysarthria
HP:0001263	Global developmental delay
HP:0001272	Cerebellar atrophy
HP:0001284	Areflexia
HP:0001337	Tremor

GWAS associations

3 associations (top):

Study	Trait	p-value
GCST004224_15	Coronary atherosclerosis (increased number of diseased vessels) (traffic exposure interaction)	5.000000e-06
GCST006624_89	Systolic blood pressure	5.000000e-09
GCST012305_12	Major depressive disorder x sex interaction	6.000000e-06

EFO canonical traits (4, from GWAS)

EFO ID	Trait name
EFO:0007908	traffic air pollution measurement
EFO:0007938	coronary atherosclerosis measurement
EFO:0006335	systolic blood pressure
EFO:0008343	sex interaction measurement

MeSH disease descriptors (3)

Descriptor	Name	Tree numbers
D001321	Autistic Disorder	F03.625.164.113.500
D002524	Cerebellar Ataxia	C10.228.140.252.190; C10.597.350.090.500; C23.888.592.350.090.200
D020190	Myoclonic Epilepsy, Juvenile	C10.228.140.490.375.130.670; C10.228.140.490.493.063.670

Drugs & pharmacology

Drug and pharmacology data

Is drug target: yes

ChEMBL targets (1): CHEMBL4295821 (SINGLE PROTEIN)

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

ChEMBL bioactivities

2 potent at pChembl≥5 of 2 total, top 2 by pChembl (potency: 10 = 0.1 nM, 6 = 1 µM).

pChembl	Type	Value	Unit	Molecule
6.30	Kd	497.5	nM	CHEMBL5653589
6.30	ED50	497.5	nM	CHEMBL5653589

PubChem BioAssay actives

1 with measured affinity, of 3 total; 1 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	2147994: Binding affinity to human CAPRIN1 incubated for 45 mins by Kinobead based pull down assay	kd	0.4975	uM

CTD chemical–gene interactions

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

Chemical	Actions (top 5)	PubMed papers
Valproic Acid	decreases expression, affects expression, affects cotreatment	6
sodium arsenite	decreases expression, affects localization, decreases reaction, affects binding, increases reaction	4
bisphenol A	affects expression, decreases expression, increases expression	3
Ozone	affects expression, affects cotreatment, decreases expression, increases oxidation, increases abundance	3
methacrylaldehyde	affects cotreatment, decreases expression, increases oxidation, increases abundance	2
4-(5-benzo(1,3)dioxol-5-yl-4-pyridin-2-yl-1H-imidazol-2-yl)benzamide	affects cotreatment, decreases expression	2
Acrolein	affects cotreatment, decreases expression, increases oxidation, increases abundance	2
Air Pollutants	increases oxidation, affects expression, affects cotreatment, decreases expression, increases abundance	2
Cadmium Chloride	decreases reaction, increases abundance, increases palmitoylation, decreases expression	2
Particulate Matter	increases expression, increases abundance	2
aristolochic acid I	decreases expression	1
bisphenol F	increases expression	1
methylmercuric chloride	decreases expression	1
triphenyl phosphate	affects expression	1
alpha-pinene	affects cotreatment, decreases expression, increases oxidation, increases abundance	1
pyrogallol 1,3-dimethyl ether	affects localization, increases expression, affects cotreatment	1
trichostatin A	affects expression	1
arsenite	increases reaction, affects binding	1
cobaltous chloride	decreases expression	1
butyraldehyde	decreases expression	1
2-bromopalmitate	decreases reaction, increases abundance, increases palmitoylation	1
perfluorooctanoic acid	decreases expression	1
nivalenol	increases expression	1
beta-methylcholine	affects expression	1
pentanal	decreases expression	1
tamibarotene	decreases expression	1
2,3,5-(triglutathion-S-yl)hydroquinone	decreases ADP-ribosylation	1
belinostat	decreases expression	1
bisphenol B	increases expression	1
14-deoxy-11,12-didehydroandrographolide	increases expression	1

ChEMBL screening assays

2 unique, capped per target: 2 binding

Representative assays (with source publication via chembl_document):

Assay ID	Type	Description	Source paper
CHEMBL4119046	Binding	Binding affinity to CAPRIN1 in human NCI-H358 cells at 1 uM by mass spectrometry based pull down assay	Studies of TAK1-centered polypharmacology with novel covalent TAK1 inhibitors. — Bioorg Med Chem

Cellosaurus cell lines

2 cell lines: 1 cancer cell line, 1 induced pluripotent stem cell

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

Cellosaurus	Name	Category	Sex
CVCL_D7LN	Ubigene A-549 CAPRIN1 KO	Cancer cell line	Male
CVCL_E8AZ	IGGi006-A	Induced pluripotent stem cell	Female

Clinical trials (associated diseases)

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

Trial	Phase	Status	Title
NCT00391261	PHASE4	COMPLETED	An Open-label Trial of Metformin for Weight Control of Pediatric Patients on Antipsychotic Medications.
NCT01028820	PHASE4	COMPLETED	FMRI Brain Activation of Aripiprazole Treatment in Autism Spectrum Disorders
NCT01333865	PHASE4	COMPLETED	A Study of Memantine Hydrochloride (Namenda®) for Cognitive and Behavioral Impairment in Adults With Autism Spectrum Disorders
NCT01337700	PHASE4	COMPLETED	Milnacipran in Autism and the Functional Locus Coeruleus and Noradrenergic Model of Autism
NCT01695200	PHASE4	COMPLETED	Omega-3 Fatty Acids in Autism Spectrum Disorders
NCT02096952	PHASE4	COMPLETED	Methylphenidate ER Liquid Formulation in Adults With ASD and ADHD
NCT02235467	PHASE4	COMPLETED	Multisite Study: Parental Training Using Video Modelling to Develop Social Skills in Children With Autism
NCT02940574	PHASE4	COMPLETED	Neural and Behavioral Effects of Oxytocin in Autism Spectrum Disorders
NCT03333629	PHASE4	COMPLETED	Promoting Positive Outcomes for Individuals With ASD: Linking Early Detection, Treatment, and Long-term Outcomes
NCT03337646	PHASE4	COMPLETED	Evaluation of the Effect and Safety of Lisdexamfetamine in Children Aged 6-12 With ADHD and Autism
NCT03538431	PHASE4	COMPLETED	Improving Driving in Young People With Autism Spectrum Disorders
NCT03757585	PHASE4	COMPLETED	Natural Treatments for the Management of Emotional Dysregulation in Youth With Non-verbal Learning Disability (NVLD) and/or Autism Spectrum Disorders (ASD)
NCT04903353	PHASE4	COMPLETED	Pragmatic Trial Comparing Weight Gain in Children With Autism Taking Risperidone Versus Aripiprazole
NCT05063656	PHASE4	COMPLETED	Biomarker-Driven Pharmacological Treatment of Adolescents With Autism Spectrum Disorder With Gabapentin
NCT05146245	PHASE4	UNKNOWN	Safety and Pharmacokinetics of Antipsychotics in Children 2: Studying TDM in an RCT
NCT05916339	PHASE4	RECRUITING	AWARE: Management of ADHD in Autism Spectrum Disorder
NCT05954052	PHASE4	TERMINATED	A Study of Glutathione in Children With Autism Spectrum Disorder
NCT06853665	PHASE4	RECRUITING	The TEAM Study - Treatment Efficacy for Autism/Attention Using Mixed Amphetamine
NCT07054697	PHASE4	COMPLETED	Pilot-RCT With Individualized Homeopathic Treatment in the Children With Autism Spectrum Disorder
NCT07161804	PHASE4	COMPLETED	Pilot RCT Using Homeopathic Medicines in ASD
NCT07439042	PHASE4	NOT_YET_RECRUITING	Buspirone for Anxiety in Autistic Youth
NCT01302964	PHASE3	COMPLETED	Mirtazapine Treatment of Anxiety in Children and Adolescents With Pervasive Developmental Disorders
NCT01706523	PHASE3	TERMINATED	Open Label Extension Study of STX209 (Arbaclofen) in Autism Spectrum Disorders
NCT01825798	PHASE3	COMPLETED	Treatment of Overweight Induced by Antipsychotic Medication in Young People With Autism Spectrum Disorders (ASD)
NCT01972074	PHASE3	COMPLETED	Behavioral and Neural Response to Memantine in Adolescents With Autism Spectrum Disorder
NCT02985749	PHASE3	COMPLETED	A Study of Oxytocin for the Treatment of Social Impairment in Individuals With High Functioning Autism Spectrum Disorder
NCT03197922	PHASE3	COMPLETED	Treatment of Encopresis in Children With Autism Spectrum Disorders
NCT03504917	PHASE3	TERMINATED	A Study of Balovaptan in Adults With Autism Spectrum Disorder With a 2-Year Open-Label Extension
NCT03553875	PHASE3	TERMINATED	Memantine for the Treatment of Social Deficits in Youth With Disorders of Impaired Social Interactions
NCT03640156	PHASE3	COMPLETED	Modulating Socially Adaptive Mirror System Functioning in Autism by Oxytocin
NCT03715153	PHASE3	TERMINATED	Efficacy and Safety of Bumetanide Oral Liquid Formulation in Children Aged From 2 to Less Than 7 Years Old With Autism Spectrum Disorder.
NCT03715166	PHASE3	TERMINATED	Efficacy and Safety of Bumetanide Oral Liquid Formulation in Children and Adolescents Aged From 7 to Less Than 18 Years Old With Autism Spectrum Disorder
NCT04233502	PHASE3	WITHDRAWN	Efficacy and Safety of Slenyto for Insomnia in Children With ASD
NCT04578756	PHASE3	COMPLETED	Open-Label, Flexible-dose Study to Evaluate the Long-Term Safety and Tolerability of Cariprazine in the Treatment of Pediatric Participants With Schizophrenia, Bipolar I Disorder, or Autism Spectrum Disorder
NCT04623398	PHASE3	COMPLETED	Effect of Lithium in Patients With Autism Spectrum Disorder and Phelan-McDermid Syndrome (SHANK3 Haploinsufficiency)
NCT04725383	PHASE3	TERMINATED	Amitriptyline for Repetitive Behaviors in Autism Spectrum Disorders
NCT05212493	PHASE3	COMPLETED	The Effects of Medical Cannabis in Children With Autistic Spectrum Disorder
NCT05361707	PHASE3	UNKNOWN	Evaluating the Effects of Tasimelteon in Individuals With Autism Spectrum Disorder (ASD) and Sleep Disturbances
NCT05439616	PHASE3	COMPLETED	Study of Cariprazine Oral Capsules or Solution to Assess Adverse Events and Change in Irritability Due to Autism Spectrum Disorder (ASD) in Participants Aged 5-17 Years With ASD
NCT06229210	PHASE3	RECRUITING	Safety and Tolerability Trial of Lumateperone in Pediatric Patients With Schizophrenia, Bipolar Disorder or Autism Spectrum Disorder