Neurodevelopmental disorder with or without hyperkinetic movements and seizures, autosomal dominant

disease
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Also known as autosomal dominant intellectual disability 8autosomal dominant non-syndromic intellectual disability caused by mutation in GRIN1GRIN1 autosomal dominant non-syndromic intellectual disabilityintellectual disability, autosomal dominant 8intellectual disability, autosomal dominant type 8mental retardation, autosomal dominant 8mental retardation, autosomal dominant 8, formerlymental retardation, autosomal dominant type 8MRD8NDHMSD

Summary

Neurodevelopmental disorder with or without hyperkinetic movements and seizures, autosomal dominant (MONDO:0013655) is a disease caused by GRIN1 (GenCC Definitive), with 4 cohort genes.

At a glance

  • Causal gene: GRIN1 (GenCC Definitive)
  • Cohort genes: 4
  • ClinVar variants: 1,031

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

FieldValue
Canonical nameneurodevelopmental disorder with or without hyperkinetic movements and seizures, autosomal dominant
Mondo IDMONDO:0013655
OMIM614254
DOIDDOID:0070038
UMLSC3280282
MedGen481912
GARD0013686
Is cancer (heuristic)no

Also known as: autosomal dominant intellectual disability 8 · autosomal dominant non-syndromic intellectual disability caused by mutation in GRIN1 · GRIN1 autosomal dominant non-syndromic intellectual disability · intellectual disability, autosomal dominant 8 · intellectual disability, autosomal dominant type 8 · mental retardation, autosomal dominant 8 · mental retardation, autosomal dominant 8, formerly · mental retardation, autosomal dominant type 8 · MRD8 · NDHMSD · neurodevelopmental disorder with or without hyperkinetic movements and seizures, autosomal dominant

Data availability: 1,031 ClinVar variants · 4 GenCC gene-disease records · 1 cell line.

Disease family

Classification path: disease › human disease › disease by etiologic mechanism › disease of genetic or genomic mechanism › hereditary disease › autosomal genetic disease › autosomal dominant disease › intellectual disability, autosomal dominantneurodevelopmental disorder with or without hyperkinetic movements and seizures, autosomal dominant

Related subtypes (28): intellectual disability, autosomal dominant 1, intellectual disability, autosomal dominant 3, intellectual disability, autosomal dominant 4, intellectual disability, autosomal dominant 5, intellectual disability, autosomal dominant 6, intellectual disability, autosomal dominant 2, intellectual disability, autosomal dominant 9, intellectual disability, autosomal dominant 10, intellectual disability, autosomal dominant 11, intellectual disability, autosomal dominant 24, intellectual disability, autosomal dominant 38, intellectual disability, autosomal dominant 39, intellectual disability, autosomal dominant 40, intellectual disability, autosomal dominant 42, autosomal dominant non-syndromic intellectual disability, intellectual developmental disorder, autosomal dominant 65, intellectual developmental disorder, autosomal dominant 69, intellectual developmental disorder, autosomal dominant 66, intellectual developmental disorder, autosomal dominant 64, intellectual developmental disorder, autosomal dominant 67, intellectual developmental disorder, autosomal dominant 68, autosomal dominant syndromic intellectual disability, intellectual developmental disorder, autosomal dominant 70, intellectual developmental disorder, autosomal dominant 71, with behavioral abnormalities, intellectual developmental disorder, autosomal dominant 72, intellectual developmental disorder, autosomal dominant 74, intellectual developmental disorder, autosomal dominant 75, intellectual developmental disorder, autosomal dominant 76

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

ClinVar germline variants

600 retrieved; paginated sample, class counts are floors:

335 likely benign, 164 uncertain significance, 26 conflicting classifications of pathogenicity, 22 benign, 22 likely pathogenic, 21 pathogenic, 6 benign/likely benign, 4 pathogenic/likely pathogenic

ClinVarVariant (HGVS)GeneClassificationReview
1076783NM_007327.4(GRIN1):c.1921A>G (p.Met641Val)GRIN1Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
1284989NM_007327.4(GRIN1):c.1976G>A (p.Arg659Gln)GRIN1Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
1329920NM_007327.4(GRIN1):c.1950C>A (p.Asn650Lys)GRIN1Pathogeniccriteria provided, single submitter
1445348NM_007327.4(GRIN1):c.2438T>C (p.Met813Thr)GRIN1Pathogeniccriteria provided, single submitter
1452432NM_007327.4(GRIN1):c.2443G>C (p.Gly815Arg)GRIN1Pathogeniccriteria provided, single submitter
1483551NM_007327.4(GRIN1):c.1324_1325dup (p.Ser443fs)GRIN1Pathogeniccriteria provided, single submitter
1484756NM_007327.4(GRIN1):c.529C>T (p.Gln177Ter)GRIN1Pathogeniccriteria provided, single submitter
1685863NM_007327.4(GRIN1):c.1668G>T (p.Gln556His)GRIN1Pathogeniccriteria provided, single submitter
1685864NM_007327.4(GRIN1):c.1832G>T (p.Trp611Leu)GRIN1Pathogeniccriteria provided, single submitter
1700197NM_007327.4(GRIN1):c.1921A>T (p.Met641Leu)GRIN1Pathogeniccriteria provided, multiple submitters, no conflicts
1713267NM_007327.4(GRIN1):c.1665G>T (p.Met555Ile)GRIN1Pathogeniccriteria provided, single submitter
1922738NM_007327.4(GRIN1):c.1069C>T (p.Gln357Ter)GRIN1Pathogeniccriteria provided, single submitter
1949360NM_007327.4(GRIN1):c.1078A>T (p.Lys360Ter)GRIN1Pathogeniccriteria provided, single submitter
2000563NM_007327.4(GRIN1):c.2030_2034del (p.Asp677fs)GRIN1Pathogeniccriteria provided, single submitter
208743NM_007327.4(GRIN1):c.2443G>A (p.Gly815Arg)GRIN1Pathogeniccriteria provided, multiple submitters, no conflicts
209159NM_007327.4(GRIN1):c.1858G>C (p.Gly620Arg)GRIN1Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
2097493NM_007327.4(GRIN1):c.1798_1805del (p.Ala600fs)GRIN1Pathogeniccriteria provided, single submitter
2119508NM_007327.4(GRIN1):c.118C>T (p.Gln40Ter)GRIN1Pathogeniccriteria provided, single submitter
2147758NM_007327.4(GRIN1):c.268del (p.Ile90fs)GRIN1Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
2499551NM_007327.4(GRIN1):c.2451C>A (p.Phe817Leu)GRIN1Pathogeniccriteria provided, single submitter
2836372NM_007327.4(GRIN1):c.1736del (p.Leu579fs)GRIN1Pathogeniccriteria provided, single submitter
2866137NM_007327.4(GRIN1):c.1214A>T (p.Gln405Leu)GRIN1Pathogeniccriteria provided, single submitter
2910238NM_007327.4(GRIN1):c.2365G>A (p.Asp789Asn)GRIN1Pathogeniccriteria provided, single submitter
29725NM_007327.4(GRIN1):c.1984G>A (p.Glu662Lys)GRIN1Pathogenicno assertion criteria provided
29726NM_007327.4(GRIN1):c.1679_1681dup (p.Ser560dup)GRIN1Pathogenicno assertion criteria provided
1333808NM_007327.4(GRIN1):c.1850C>T (p.Ser617Phe)GRIN1Likely pathogeniccriteria provided, single submitter
1481271NM_007327.4(GRIN1):c.2380C>G (p.Arg794Gly)GRIN1Likely pathogeniccriteria provided, single submitter
1679134NM_007327.4(GRIN1):c.1664T>C (p.Met555Thr)GRIN1Likely pathogeniccriteria provided, single submitter
1706588NM_007327.4(GRIN1):c.2377G>T (p.Val793Phe)GRIN1Likely pathogeniccriteria provided, single submitter
1710175NM_007327.4(GRIN1):c.2171+1G>AGRIN1Likely pathogenicno assertion criteria provided

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 10 · Orphanet: 5 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

GenCC gene–disease validity (cohort genes)

the Disease column is the GenCC-asserted condition — a cohort gene’s strongest validity may be for a related predisposition syndrome.

GeneClassificationInheritanceDiseaseRecords
GRIN1DefinitiveAutosomal dominantcomplex neurodevelopmental disorder10

Orphanet rare-disease linkage (cohort genes)

GeneOrphanet IDRare disease
GRIN1Orphanet:178469Autosomal dominant non-syndromic intellectual disability
GRIN1Orphanet:1934Early infantile developmental and epileptic encephalopathy
GRIN1Orphanet:208447Bilateral generalized polymicrogyria
GRIN1Orphanet:88616Autosomal recessive non-syndromic intellectual disability
ABCA2Orphanet:88616Autosomal recessive non-syndromic intellectual disability

Cohort genes → proteins

4 cohort genes, 4 distinct canonical proteins.

Evidence partition

SubsetGenes
multi_evidence4

Cohort genes (full)

SymbolHGNCEnsemblUniProtNameEvidence
GRIN1HGNC:4584ENSG00000176884Q05586Glutamate receptor ionotropic, NMDA 1gencc,clinvar
DPP7HGNC:14892ENSG00000176978Q9UHL4Dipeptidyl peptidase 2clinvar
ANAPC2HGNC:19989ENSG00000176248Q9UJX6Anaphase-promoting complex subunit 2clinvar
ABCA2HGNC:32ENSG00000107331Q9BZC7ATP-binding cassette sub-family A member 2clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

SymbolProtein nameFunction (lead sentence)
GRIN1Glutamate receptor ionotropic, NMDA 1Component of N-methyl-D-aspartate (NMDA) receptors (NMDARs) that function as heterotetrameric, ligand-gated cation channels with high calcium permeability and voltage-dependent block by Mg(2+).
DPP7Dipeptidyl peptidase 2Plays an important role in the degradation of some oligopeptides.
ANAPC2Anaphase-promoting complex subunit 2Together with the RING-H2 protein ANAPC11, constitutes the catalytic component of the anaphase promoting complex/cyclosome (APC/C), a cell cycle-regulated E3 ubiquitin ligase that controls progression through mitosis and the G1 phase of th…
ABCA2ATP-binding cassette sub-family A member 2Probable lipid transporter that modulates cholesterol sequestration in the late endosome/lysosome by regulating the intracellular sphingolipid metabolism, in turn participates in cholesterol homeostasis.

Protein-family classification

Druggable: 2 · Difficult: 0 · Unknown: 2 · Druggable fraction: 0.5

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

FamilyGenesFoldFDR
Transporter119.4×0.151
Protease19.2×0.157
Other/Unknown20.9×0.769

Per-gene assignment

SymbolFamilyDruggable?ECInterPro (top 3)
GRIN1Other/UnknownnoIontro_rcpt_C, Iono_Glu_rcpt_met, ANF_lig-bd_rcpt
DPP7Proteaseyes3.4.14.2Peptidase_S28, AB_hydrolase_fold, Ser_carbopepase_S28_SKS
ANAPC2Other/UnknownnoANAPC2_C, Cullin_homology, Cullin_homology_sf
ABCA2TransporteryesABC_transporter-like_ATP-bd, AAA+_ATPase, ABC2_TM

Expression context

Cohort genes with no expression data: 0.

3 cohort genes are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

BucketGenes
narrow (1-5 tissues)0
moderate (6-20)0
broad (>20)4
unknown0

Top tissues across cohort

TissueCohort genes
right hemisphere of cerebellum4
right uterine tube2
cerebellar hemisphere1
right frontal lobe1
adenohypophysis1
left testis1
C1 segment of cervical spinal cord1
spinal cord1

Per-gene tissue summary (top 30)

SymbolBgee breadthFANTOM5 breadthSCXATop tissues
GRIN1215tissue_specificmarkerright hemisphere of cerebellum, right frontal lobe, cerebellar hemisphere
DPP7243ubiquitousmarkeradenohypophysis, right hemisphere of cerebellum, right uterine tube
ANAPC2237ubiquitousyesright uterine tube, right hemisphere of cerebellum, left testis
ABCA2234ubiquitousmarkerC1 segment of cervical spinal cord, spinal cord, right hemisphere of cerebellum

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

SymbolInteractor count
ANAPC22,288
ABCA21,678
DPP71,078
GRIN1118

Structural data

PDB: 3 · AlphaFold-only: 1 · No structure: 0

Cohort genes with PDB structures (top 30)

SymbolUniProtPDB entries
GRIN1Q0558685
ANAPC2Q9UJX623
DPP7Q9UHL43

AlphaFold-only cohort genes (top 30 by pLDDT)

SymbolUniProtpLDDT
ABCA2Q9BZC771.46

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 60. Enrichment computed across 4 evidence-associated genes (4 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 4 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

PathwayCohort genesFoldFDRSample cohort genes
Inhibition of the proteolytic activity of APC/C required for the onset of anaphase by mitotic spindle checkpoint components1158.6×0.033ANAPC2
ABC transporters in lipid homeostasis1150.3×0.033ABCA2
Ras activation upon Ca2+ influx through NMDA receptor1142.8×0.033GRIN1
Phosphorylation of the APC/C1135.9×0.033ANAPC2
Unblocking of NMDA receptors, glutamate binding and activation1135.9×0.033GRIN1
Synaptic adhesion-like molecules1135.9×0.033GRIN1
Negative regulation of NMDA receptor-mediated neuronal transmission1135.9×0.033GRIN1
Inactivation of APC/C via direct inhibition of the APC/C complex1129.8×0.033ANAPC2
Conversion from APC/C:Cdc20 to APC/C:Cdh1 in late anaphase1129.8×0.033ANAPC2
Aberrant regulation of mitotic exit in cancer due to RB1 defects1129.8×0.033ANAPC2
Long-term potentiation1119.0×0.033GRIN1
APC/C:Cdc20 mediated degradation of Cyclin B1114.2×0.033ANAPC2
APC-Cdc20 mediated degradation of Nek2A1105.7×0.033ANAPC2
APC:Cdc20 mediated degradation of cell cycle proteins prior to satisfation of the cell cycle checkpoint1105.7×0.033ANAPC2
Activation of APC/C and APC/C:Cdc20 mediated degradation of mitotic proteins1102.0×0.033ANAPC2
Aberrant regulation of mitotic cell cycle due to RB1 defects1102.0×0.033ANAPC2
Diseases of mitotic cell cycle198.5×0.033ANAPC2
APC/C:Cdc20 mediated degradation of mitotic proteins189.2×0.033ANAPC2
APC/C-mediated degradation of cell cycle proteins184.0×0.033ANAPC2
Regulation of mitotic cell cycle184.0×0.033ANAPC2
DNA Replication Pre-Initiation179.3×0.033ANAPC2
Regulation of APC/C activators between G1/S and early anaphase177.2×0.033ANAPC2
Switching of origins to a post-replicative state175.1×0.033ANAPC2
Synthesis of DNA175.1×0.033ANAPC2
EPHB-mediated forward signaling166.4×0.035GRIN1
Transcriptional Regulation by VENTX166.4×0.035ANAPC2
Assembly and cell surface presentation of NMDA receptors163.4×0.035GRIN1
DNA Replication159.5×0.036ANAPC2
Neurexins and neuroligins149.2×0.040GRIN1
Autodegradation of Cdh1 by Cdh1:APC/C147.6×0.040ANAPC2

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 4 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO termCohort genesFoldFDRSample cohort genes
propylene metabolic process14213.0×0.003GRIN1
negative regulation of intracellular cholesterol transport14213.0×0.003ABCA2
negative regulation of phospholipid biosynthetic process14213.0×0.003ABCA2
negative regulation of sphingolipid biosynthetic process14213.0×0.003ABCA2
regulation of protein localization to cell periphery14213.0×0.003ABCA2
response to glycine14213.0×0.003GRIN1
negative regulation of steroid metabolic process12106.5×0.003ABCA2
ceramide translocation12106.5×0.003ABCA2
regulation of post-translational protein modification12106.5×0.003ABCA2
negative regulation of receptor-mediated endocytosis involved in cholesterol transport12106.5×0.003ABCA2
positive regulation of low-density lipoprotein particle receptor catabolic process11404.3×0.005ABCA2
ganglioside metabolic process11053.2×0.005ABCA2
regulation of intracellular cholesterol transport11053.2×0.005ABCA2
sphingomyelin metabolic process1842.6×0.005ABCA2
positive regulation of amyloid precursor protein biosynthetic process1842.6×0.005ABCA2
intracellular sphingolipid homeostasis1842.6×0.005ABCA2
negative regulation of cholesterol efflux1702.2×0.006ABCA2
glycosphingolipid metabolic process1601.9×0.006ABCA2
regulation of steroid metabolic process1601.9×0.006ABCA2
central nervous system myelin formation1601.9×0.006ABCA2
metaphase/anaphase transition of mitotic cell cycle1526.6×0.006ANAPC2
regulation of monoatomic cation transmembrane transport1526.6×0.006GRIN1
positive regulation of synapse maturation1468.1×0.007ANAPC2
response to cholesterol1421.3×0.007ABCA2
positive regulation of amyloid precursor protein catabolic process1421.3×0.007ABCA2
regulation of protein localization to cell surface1421.3×0.007ABCA2
positive regulation of synaptic plasticity1383.0×0.007ANAPC2
calcium ion transmembrane import into cytosol1383.0×0.007GRIN1
ionotropic glutamate receptor signaling pathway1324.1×0.008GRIN1
excitatory chemical synaptic transmission1324.1×0.008GRIN1

Therapeutics

Drug target analysis

Approved (phase 4): 2 · Phase ≥3: 2 · Phased (≥1): 2 · Undrugged: 2

Druggability breadth: 2 of 4 evidence-associated genes (50%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Genes with an approved drug

The molecule shown is one approved compound that hits the gene — not necessarily a drug of choice or one indicated for this disease.

SymbolExample approved molecule
GRIN1DEXTROMETHORPHAN
DPP7SITAGLIPTIN

Top cohort targets by molecule count

SymbolMoleculesMax phase
GRIN1394
DPP734
ANAPC200
ABCA200

Drugs targeting cohort genes (top 30)

MoleculeMax phaseTargets in cohort
DEXTROMETHORPHAN4GRIN1
KETAMINE4GRIN1
CYCLOSERINE4GRIN1
MEMANTINE4GRIN1
TACRINE4GRIN1
MEMANTINE HYDROCHLORIDE4GRIN1
ESKETAMINE4GRIN1
PENTAMIDINE4GRIN1
AMANTADINE4GRIN1
LEVORPHANOL4GRIN1
CHLORPROMAZINE4GRIN1
PROCYCLIDINE4GRIN1
ORPHENADRINE4GRIN1
SITAGLIPTIN4DPP7
GOSOGLIPTIN4DPP7
ESMETHADONE3GRIN1
DALZANEMDOR3GRIN1
LATREPIRDINE3GRIN1
GLUTAMIC ACID3GRIN1
TALABOSTAT3DPP7
DEXTRORPHAN2GRIN1
LEVOMETHADONE2GRIN1
ALPHAMETHADOL2GRIN1
TRAXOPRODIL2GRIN1
RADIPRODIL2GRIN1
PHENCYCLIDINE2GRIN1
DIZOCILPINE2GRIN1
ELIPRODIL2GRIN1
IFENPRODIL2GRIN1
ONFASPRODIL2GRIN1

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 1.

Cohort genes with ChEMBL bioactivity (full, sorted by assay count)

SymbolAssaysType breakdown
GRIN1481Binding:435, Functional:40, ADMET:5, Toxicity:1
DPP7147Binding:142, Functional:4, ADMET:1

Cohort enzymes (BRENDA EC)

SymbolEC numbersNames
DPP73.4.14.2dipeptidyl-peptidase II

Cohort genes with high screening signal

≥100 ChEMBL assays — a studied-ness signal; see Therapeutics for approved-drug status.

SymbolChEMBL assays
GRIN1481
DPP7147

Pharmacogenomics

Cohort genes with a PharmGKB record: 4; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Chemical tractability of cohort targets

30 approved/phased compounds have measured bioactivity against a cohort gene (and aren’t yet in disease-level trials). This is a research / tractability signal, NOT a therapeutic recommendation — a bioactivity row often reflects off-target or screening binding (e.g. promiscuous kinase inhibitors against a cohort kinase), implying no disease mechanism.

CompoundMax phaseCohort target (bioactivity)
DEXTROMETHORPHAN4GRIN1
KETAMINE4GRIN1
CYCLOSERINE4GRIN1
MEMANTINE4GRIN1
TACRINE4GRIN1
MEMANTINE HYDROCHLORIDE4GRIN1
ESKETAMINE4GRIN1
PENTAMIDINE4GRIN1
AMANTADINE4GRIN1
LEVORPHANOL4GRIN1
CHLORPROMAZINE4GRIN1
PROCYCLIDINE4GRIN1
ORPHENADRINE4GRIN1
SITAGLIPTIN4DPP7
GOSOGLIPTIN4DPP7
ESMETHADONE3GRIN1
DALZANEMDOR3GRIN1
LATREPIRDINE3GRIN1
GLUTAMIC ACID3GRIN1
TALABOSTAT3DPP7
DEXTRORPHAN2GRIN1
LEVOMETHADONE2GRIN1
ALPHAMETHADOL2GRIN1
TRAXOPRODIL2GRIN1
RADIPRODIL2GRIN1
PHENCYCLIDINE2GRIN1
DIZOCILPINE2GRIN1
ELIPRODIL2GRIN1
IFENPRODIL2GRIN1
ONFASPRODIL2GRIN1

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

TierDefinitionGenesSymbols
AApproved (phase 4 drug)2GRIN1, DPP7
BPhased (≥1) drug, not yet approved0
CDruggable family + PDB, no drug0
DDruggable family + AlphaFold only, no drug1ABCA2
EDifficult family or no structure, no drug1ANAPC2

Undrugged target profiles

2 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

SymbolChEMBL assaysDrugged partners (top 3)
ANAPC20
ABCA20

Clinical trials & evidence

Clinical trials

Clinical trials: 0.