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GeneE
10 sources retrieved · Most recent: April 2026 · Index updated 14 days ago
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SPTBN4
spectrin beta, non-erythrocytic 4
Chromosome 19 · 19q13.2
NCBI Gene: 57731Ensembl: ENSG00000160460.17HGNC: HGNC:14896UniProt: Q9H254
44PubMed Papers
21Diseases
0Drugs
33Pathogenic Variants
CLINICAL
OMIM Disease Gene
DATA QUALITY
✓ Experimental GO Evidence✓ Swiss-Prot Reviewed
membraneextracellular exosomePML bodyphosphatase bindingneurodevelopmental disorder with hypotonia, neuropathy, and deafnessperipheral neuropathy-myopathy-hoarseness-hearing loss syndromegenetic disorderneurodegenerative disease
✦AI Summary

SPTBN4 encodes spectrin beta, non-erythrocytic 4, a cytoskeletal protein essential for neuronal organization and function. As a non-erythrocytic β-spectrin, SPTBN4 assembles with α-spectrin subunits to form heterodimers that bind to cytoskeletal elements and the plasma membrane, enabling proper localization of ion channels, signal transduction, and cellular scaffolding 1. SPTBN4 plays specialized roles in axonal organization, including clustering of voltage-gated sodium channels at the node of Ranvier and maintenance of axonal domains critical for neuronal function 2. Pathogenic variants in SPTBN4 cause neurodevelopmental disorder with hypotonia, neuropathy, and deafness (NEDHND), an autosomal recessive condition 3. Affected individuals present with severe muscular hypotonia, dysphagia, absent or delayed speech, gross motor and mental retardation, with variable features including nystagmus, epileptiform EEG discharges, and choreoathetosis 3. Muscle histology reveals both myopathic and neuropathic characteristics 3. However, clinical presentation varies; some patients exhibit primarily axonal neuropathy with hypotonia without intellectual disability or seizures 4. Animal models demonstrate that SPTBN4 deficiency causes severe myopathy, hind-limb paralysis, and tremors 5. These findings establish SPTBN4 as critical for maintaining neuronal cytoskeletal integrity and proper axonal function.

Sources cited
1
Spectrins bind cytoskeletal elements and plasma membrane for protein localization and cellular scaffolding; SPTBN4 pathogenic variants are associated with neurological disorders
PMID: 33847457
2
SPTBN4 is a neuronal spectrin with specialized roles in axonal organization and causes neurological spectrinopathies
PMID: 36697767
3
SPTBN4 variants cause NEDHND with hypotonia, neuropathy, intellectual disability, dysphagia, seizures, and muscle histology showing myopathic and neuropathic features
PMID: 33772159
4
SPTBN4 mutations can present with axonal neuropathy and hypotonia without intellectual disability or feeding difficulties
PMID: 31857255
5
SPTBN4 frameshift deletion in animals causes severe myopathy, hind-limb paralysis, tremors, and muscle fiber degeneration
PMID: 31850074
Disease Associationsⓘ21
neurodevelopmental disorder with hypotonia, neuropathy, and deafnessOpen Targets
0.78Strong
peripheral neuropathy-myopathy-hoarseness-hearing loss syndromeOpen Targets
0.55Moderate
genetic disorderOpen Targets
0.51Moderate
neurodegenerative diseaseOpen Targets
0.36Weak
deafnessOpen Targets
0.12Weak
hearing loss, autosomal recessiveOpen Targets
0.11Weak
autosomal dominant nonsyndromic hearing lossOpen Targets
0.11Weak
Global developmental delayOpen Targets
0.11Weak
Delayed myelinationOpen Targets
0.11Weak
Flexion contractureOpen Targets
0.11Weak
HypotoniaOpen Targets
0.11Weak
essential tremorOpen Targets
0.10Weak
movement disorderOpen Targets
0.09Suggestive
Early-onset X-linked optic atrophyOpen Targets
0.09Suggestive
optic atrophy 2Open Targets
0.09Suggestive
dystonia 27Open Targets
0.09Suggestive
spinocerebellar ataxia type 15/16Open Targets
0.09Suggestive
Spinocerebellar ataxia type 40Open Targets
0.08Suggestive
Young adult-onset ParkinsonismOpen Targets
0.08Suggestive
X-linked dystonia-parkinsonismOpen Targets
0.08Suggestive
Neurodevelopmental disorder with hypotonia, neuropathy, and deafnessUniProt
Pathogenic Variants33
NM_020971.3(SPTBN4):c.1090C>T (p.Gln364Ter)Likely pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2025→ Residue 364
NM_020971.3(SPTBN4):c.2265G>A (p.Trp755Ter)Pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2024→ Residue 755
NM_020971.3(SPTBN4):c.2535_2554del (p.Gly846fs)Likely pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2024→ Residue 846
NM_020971.3(SPTBN4):c.1217T>C (p.Leu406Pro)Likely pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2024→ Residue 406
NM_020971.3(SPTBN4):c.2903+2T>CLikely pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2024
NM_020971.3(SPTBN4):c.3948+1G>ALikely pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2023
NM_020971.3(SPTBN4):c.6826G>T (p.Glu2276Ter)Likely pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2023→ Residue 2276
NM_020971.3(SPTBN4):c.6016C>T (p.Arg2006Ter)Pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2023→ Residue 2006
NM_020971.3(SPTBN4):c.5173C>T (p.Gln1725Ter)Pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2023→ Residue 1725
NM_020971.3(SPTBN4):c.6769C>T (p.Gln2257Ter)Likely pathogenic
SPTBN4-related disorder
★☆☆☆2023→ Residue 2257
NM_020971.3(SPTBN4):c.4960C>T (p.Gln1654Ter)Likely pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2023→ Residue 1654
NM_020971.3(SPTBN4):c.1906C>T (p.Arg636Ter)Likely pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2022→ Residue 636
NM_020971.3(SPTBN4):c.6320_6336+10delPathogenic
not provided
★☆☆☆2022
NM_020971.3(SPTBN4):c.7149del (p.Pro2384fs)Likely pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2022→ Residue 2384
NM_020971.3(SPTBN4):c.5091_5092del (p.Gln1697fs)Pathogenic
not provided
★☆☆☆2020→ Residue 1697
NM_020971.3(SPTBN4):c.3589C>T (p.Gln1197Ter)Pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2020→ Residue 1197
NM_020971.3(SPTBN4):c.737G>C (p.Arg246Pro)Likely pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2020→ Residue 246
NM_020971.3(SPTBN4):c.1249del (p.Leu417fs)Pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2020→ Residue 417
NM_020971.3(SPTBN4):c.1149dup (p.Asn384fs)Pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2020→ Residue 384
NM_020971.3(SPTBN4):c.3375_3393del (p.Asp1126fs)Pathogenic
Neurodevelopmental disorder with hypotonia, neuropathy, and deafness
★☆☆☆2020→ Residue 1126
View on ClinVar ↗
Related Genes
KCNQ2Protein interaction100%KCNQ3Protein interaction100%SCN1AProtein interaction100%SCN2AProtein interaction100%ANK1Protein interaction97%ANK2Protein interaction97%
Tissue Expression6 tissues
Brain
100%
Ovary
65%
Heart
33%
Bone Marrow
10%
Liver
9%
Lung
4%
Gene Interaction Network
Click a node to explore
SPTBN4KCNQ2KCNQ3SCN1ASCN2AANK1ANK2
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted · UniProt Q9H254
View on AlphaFold ↗
Constraintⓘ
LOEUFⓘ
0.53Moderately Constrained
pLIⓘ
0.00Tolerant
Observed/Expected LoF0.45 [0.38–0.53]
RankingsWhere SPTBN4 stands among ~20K protein-coding genes
  • #9,659of 20,598
    Most Researched44
  • #1,737of 5,498
    Most Pathogenic Variants33
  • #3,309of 17,882
    Most Constrained (LOEUF)0.53 · top quartile
Genes detectedSPTBN4
Sources retrieved10 papers
Response time—
📄 Sources
10▼
1
Heterozygous variants in SPTBN1 cause intellectual disability and autism.
PMID: 33847457
Am J Med Genet A · 2021
1.00
2
Expanding the genetic heterogeneity of intellectual disability.
PMID: 28940097
Hum Genet · 2017
0.90
3
A 20-year Clinical and Genetic Neuromuscular Cohort Analysis in Lebanon: An International Effort.
PMID: 34602496
J Neuromuscul Dis · 2022
0.80
4
Spectrins: molecular organizers and targets of neurological disorders.
PMID: 36697767
Nat Rev Neurosci · 2023
0.70
5
Detection of a Frameshift Deletion in the
PMID: 31850074
Front Genet · 2019
0.60