NKX1-1

Chr 4

NK1 homeobox 1

Also known as: HSPX153, NKX-1.1, SAX2

The protein is a transcription factor that regulates genes involved in energy homeostasis, with expression predominantly in the brainstem near serotonergic neurons. Mutations cause a severe neurodevelopmental disorder characterized by early-onset failure to thrive, lack of subcutaneous fat, and early lethality, following an autosomal recessive inheritance pattern. The gene shows moderate constraint against loss-of-function variants (pLI 0.71, LOEUF 0.72).

OMIMResearchSummary from RefSeq, UniProt
LOFmechanismLOEUF 0.72
Clinical SummaryNKX1-1
Population Constraint (gnomAD)
Moderately constrained gene (pLI 0.71) — some intolerance to loss-of-function variants.
📋
ClinVar Variants
144 unique Pathogenic / Likely Pathogenic· 14 VUS of 161 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.72LOEUF
pLI 0.715
Z-score 1.88
OE 0.00 (0.000.72)
Moderately constrained

Typical tolerance to LoF variation

Missense Constraint
0.74Z-score
OE missense 0.78 (0.640.95)
67 obs / 86.3 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.00 (0.000.72)
00.351.4
Missense OE0.78 (0.640.95)
00.61.4
Synonymous OE1.08
01.21.6
LoF obs/exp: 0 / 4.1Missense obs/exp: 67 / 86.3Syn Z: -0.41
DN
0.3395th %ile
GOF
0.3193th %ile
LOF
0.81top 5%

The highest-scoring mechanism for this gene is loss-of-function (haploinsufficiency).

LOFprediction above median

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

161 submitted variants in ClinVar

Classification Summary

Pathogenic138
Likely Pathogenic6
VUS14
Likely Benign1
Benign2
138
Pathogenic
6
Likely Pathogenic
14
VUS
1
Likely Benign
2
Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories· variant type breakdown unavailable

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
138
Likely Pathogenic
6
VUS
14
Likely Benign
1
Benign
2
Total161

Counts from ClinVar esearch · Updated hourly

View in ClinVar →

Protein Context — Lollipop Plot

NKX1-1 · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

No active trials found for this gene.

Search ClinicalTrials.gov →
Clinical Literature
Open Research Assistant →
Key Publications
Landmark & review papers · by relevance
PubMed
LncRNA Expression in CD4+ T Cells in Neurosyphilis Patients.
Liu LL et al.·Front Cell Infect Microbiol
2017Cohort
Top 1 results · since 2015Search PubMed ↗
Recent Gene-Specific Literature
Gene in title · MEDLINE · newest first
Europe PMC

No open access results found