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GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
β“˜GeneE is for informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment.
NEK1
NIMA related kinase 1
Chromosome 4 Β· 4q33
NCBI Gene: 4750Ensembl: ENSG00000137601.18HGNC: HGNC:7744UniProt: A0A8I5KUL6
98PubMed Papers
23Diseases
0Drugs
106Pathogenic Variants
FUNCTIONAL ROLE
Kinase
RESEARCH IMPACT
Variant-Rich
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
nucleoplasmcentrosomecytosolcentriolar satelliteshort-rib thoracic dysplasia 6 with or without polydactylyshort rib-polydactyly syndromeamyotrophic lateral sclerosisOrofaciodigital syndrome type 2
✦AI Summary

NEK1 (NIMA-related kinase 1) is a serine/threonine kinase with multiple cellular functions critical for neuronal health and development. The protein primarily functions in DNA damage checkpoint control and repair, phosphorylating VDAC1 in response to DNA damage to limit mitochondrial cell death 1. NEK1 is essential for cilium assembly and primary ciliary structure maintenance, with mutations causing ciliary abnormalities and impaired sonic hedgehog signaling 23. The kinase regulates metabolic pathways by phosphorylating malic enzyme 1 (ME1) at S336, counteracting acetylation-mediated activation and affecting NADPH production and lipogenesis 4. NEK1 variants are significantly associated with amyotrophic lateral sclerosis (ALS), representing one of seven novel ALS genes identified since 2014 5. Loss-of-function NEK1 mutations cause ALS through ciliary dysfunction, cell cycle re-entry, disrupted tubulin acetylation, and impaired DNA damage response 3. In ALS cohorts, NEK1 variants are found in approximately 2.6% of patients, with loss-of-function variants associated with shorter survival times and upper limb onset 67. The protein's dysfunction contributes to ALS pathogenesis through multiple mechanisms including mitochondrial alterations and calcium homeostasis disruption 3.

Sources cited
1
NEK1 functions in DNA damage checkpoint control and phosphorylates VDAC1 to limit mitochondrial cell death
PMID: 20230784
2
NEK1 is involved in cilium assembly
PMID: 21211617
3
NEK1 mutations cause primary ciliary abnormalities, cell cycle re-entry, and disrupted tubulin acetylation in ALS
PMID: 40389989
4
NEK1 phosphorylates malic enzyme 1 at S336, affecting NADPH production and lipogenesis
PMID: 31735643
5
NEK1 is one of seven novel genes associated with ALS identified since 2014
PMID: 29154141
6
NEK1 variants are found in 2.6% of ALS patients with loss-of-function variants associated with shorter survival
PMID: 36443167
7
NEK1 is among the most common mutated genes in Chinese ALS cohorts
PMID: 34544842
Disease Associationsβ“˜23
short-rib thoracic dysplasia 6 with or without polydactylyOpen Targets
0.81Strong
short rib-polydactyly syndromeOpen Targets
0.59Moderate
amyotrophic lateral sclerosisOpen Targets
0.58Moderate
Orofaciodigital syndrome type 2Open Targets
0.57Moderate
orofaciodigital syndrome type IIOpen Targets
0.52Moderate
short rib-polydactyly syndrome, Majewski typeOpen Targets
0.45Moderate
smoking initiationOpen Targets
0.42Moderate
connective tissue diseaseOpen Targets
0.41Moderate
motor neuron diseaseOpen Targets
0.40Weak
Parkinson diseaseOpen Targets
0.34Weak
asphyxiating thoracic dystrophy 3Open Targets
0.33Weak
Beemer-Langer syndromeOpen Targets
0.33Weak
Alzheimer diseaseOpen Targets
0.33Weak
neurodegenerative diseaseOpen Targets
0.32Weak
multiple sclerosisOpen Targets
0.32Weak
lysosomal storage diseaseOpen Targets
0.32Weak
placental retentionOpen Targets
0.30Weak
schizophreniaOpen Targets
0.28Weak
neuroinflammatory disorderOpen Targets
0.27Weak
duodenal ulcerOpen Targets
0.27Weak
Amyotrophic lateral sclerosis 24UniProt
Orofaciodigital syndrome 2UniProt
Short-rib thoracic dysplasia 6 with or without polydactylyUniProt
Pathogenic Variants106
NM_001199397.3(NEK1):c.2241del (p.Ala750fs)Pathogenic
Short-rib thoracic dysplasia 6 with or without polydactyly
β˜…β˜…β˜†β˜†2025β†’ Residue 750
NM_001199397.3(NEK1):c.1226G>A (p.Trp409Ter)Pathogenic
Short-rib thoracic dysplasia 6 with or without polydactyly|not provided|Mohr syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 409
NM_001199397.3(NEK1):c.1618C>T (p.Arg540Ter)Pathogenic
Short-rib thoracic dysplasia 6 with or without polydactyly|NEK1-related disorder|not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 540
NM_001199397.3(NEK1):c.481C>T (p.Arg161Ter)Pathogenic
Motor neuron disease|Short-rib thoracic dysplasia 6 with or without polydactyly|NEK1-related disorder
β˜…β˜…β˜†β˜†2025β†’ Residue 161
NM_001199397.3(NEK1):c.3107C>G (p.Ser1036Ter)Pathogenic
Short-rib thoracic dysplasia 6 with or without polydactyly|Amyotrophic lateral sclerosis, susceptibility to, 24|not provided|Amyotrophic lateral sclerosis, susceptibility to, 24;Short-rib thoracic dysplasia 6 with or without polydactyly|NEK1-related disorder
β˜…β˜…β˜†β˜†2025β†’ Residue 1036
NM_001199397.3(NEK1):c.1020+1G>ALikely pathogenic
Short-rib thoracic dysplasia 6 with or without polydactyly
β˜…β˜…β˜†β˜†2025
NM_001199397.3(NEK1):c.3337G>T (p.Glu1113Ter)Pathogenic
Short-rib thoracic dysplasia 6 with or without polydactyly|not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 1113
NM_001199397.3(NEK1):c.2588-2A>GLikely pathogenic
Short-rib thoracic dysplasia 6 with or without polydactyly|Amyotrophic lateral sclerosis, susceptibility to, 24;Short-rib thoracic dysplasia 6 with or without polydactyly|not provided
β˜…β˜…β˜†β˜†2025
NM_001199397.3(NEK1):c.1097_1098del (p.Arg366fs)Pathogenic
Short-rib thoracic dysplasia 6 with or without polydactyly|not provided
β˜…β˜…β˜†β˜†2024β†’ Residue 366
NM_001199397.3(NEK1):c.1957C>T (p.Arg653Ter)Pathogenic
Short-rib thoracic dysplasia 6 with or without polydactyly|not provided|Amyotrophic lateral sclerosis, susceptibility to, 24
β˜…β˜…β˜†β˜†2024β†’ Residue 653
NM_001199397.3(NEK1):c.1606G>T (p.Glu536Ter)Pathogenic
Short-rib thoracic dysplasia 6 with or without polydactyly|Short rib-polydactyly syndrome
β˜…β˜…β˜†β˜†2024β†’ Residue 536
NM_001199397.3(NEK1):c.214+1G>APathogenic
Motor neuron disease|Short-rib thoracic dysplasia 6 with or without polydactyly|Connective tissue disorder|NEK1-related disorder
β˜…β˜…β˜†β˜†2022
NM_001199397.3(NEK1):c.869-1G>TPathogenic
Short-rib thoracic dysplasia 6 with or without polydactyly|not provided
β˜…β˜…β˜†β˜†2022
NM_001199397.3(NEK1):c.117+1G>APathogenic
not provided|Short-rib thoracic dysplasia 6 with or without polydactyly
β˜…β˜…β˜†β˜†2021
NM_001199397.3(NEK1):c.1507dup (p.Ile503fs)Pathogenic
Short-rib thoracic dysplasia 6 with or without polydactyly
β˜…β˜†β˜†β˜†2026β†’ Residue 503
NM_001199397.3(NEK1):c.2231T>G (p.Leu744Ter)Pathogenic
Amyotrophic lateral sclerosis, susceptibility to, 24
β˜…β˜†β˜†β˜†2025β†’ Residue 744
NC_000004.12:g.169508332delPathogenic
not provided
β˜…β˜†β˜†β˜†2025
NM_001199397.3(NEK1):c.2190del (p.Asn731fs)Pathogenic
Short-rib thoracic dysplasia 6 with or without polydactyly
β˜…β˜†β˜†β˜†2025β†’ Residue 731
NM_001199397.3(NEK1):c.1140+2T>CLikely pathogenic
Short-rib thoracic dysplasia 6 with or without polydactyly
β˜…β˜†β˜†β˜†2025
NM_001199397.3(NEK1):c.142del (p.Ser48fs)Pathogenic
Short-rib thoracic dysplasia 6 with or without polydactyly
β˜…β˜†β˜†β˜†2025β†’ Residue 48
View on ClinVar β†—
Related Genes
CFAP410Protein interaction94%ALS2Protein interaction94%VAPBProtein interaction88%KIF3AProtein interaction80%CHCHD10Protein interaction72%DYNC2H1Protein interaction70%
Tissue Expression6 tissues
Heart
100%
Bone Marrow
74%
Ovary
68%
Brain
43%
Lung
31%
Liver
29%
Gene Interaction Network
Click a node to explore
NEK1CFAP410ALS2VAPBKIF3ACHCHD10DYNC2H1
PROTEIN STRUCTURE
Preparing viewer…
PDB4B9D Β· 1.90 Γ… Β· X-ray
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
0.96LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.80 [0.67–0.96]
RankingsWhere NEK1 stands among ~20K protein-coding genes
  • #4,896of 20,598
    Most Researched98 Β· top quartile
  • #731of 5,498
    Most Pathogenic Variants106 Β· top quartile
  • #9,027of 17,882
    Most Constrained (LOEUF)0.96
Genes detectedNEK1
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
Novel genes associated with amyotrophic lateral sclerosis: diagnostic and clinical implications.
PMID: 29154141
Lancet Neurol Β· 2018
1.00
2
ALS-associated C21ORF2 variant disrupts DNA damage repair, mitochondrial metabolism, neuronal excitability and NEK1 levels in human motor neurons.
PMID: 39227882
Acta Neuropathol Commun Β· 2024
0.90
3
Involvement of muscle satellite cell dysfunction in neuromuscular disorders: Expanding the portfolio of satellite cell-opathies.
PMID: 35302338
Eur J Transl Myol Β· 2022
0.80
4
HDAC8 Enhances the Function of HIF-2Ξ± by Deacetylating ETS1 to Decrease the Sensitivity of TKIs in ccRCC.
PMID: 39073752
Adv Sci (Weinh) Β· 2024
0.70
5
Mutations in NEK1 cause ciliary dysfunction as a novel pathogenic mechanism in amyotrophic lateral sclerosis.
PMID: 40389989
Mol Neurodegener Β· 2025
0.60