NHS

Chr XXLDX-linked

NHS actin remodeling regulator

Also known as: CTRCT40, CXN

The NHS protein regulates actin remodeling and cell morphology, and controls development of the eye, teeth, brain, and craniofacial structures. Mutations cause Nance-Horan syndrome and X-linked cataract, both following X-linked inheritance patterns. This gene is highly constrained against loss-of-function variants (pLI 1.0, LOEUF 0.087), indicating that mutations typically have significant developmental consequences.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt
LOFmechanismXLD/X-linkedLOEUF 0.093 OMIM phenotypes
Clinical SummaryNHS
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Gene-Disease Validity (ClinGen)
Nance-Horan syndrome · XLDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 1.00). One damaged copy is likely sufficient to cause disease.
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ClinVar Variants
54 unique Pathogenic / Likely Pathogenic· 191 VUS of 500 total submissions
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Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available
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GeneReview available — NHS
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.09LOEUF
pLI 1.000
Z-score 5.42
OE 0.00 (0.000.09)
Highly constrained

Among the most LoF-intolerant genes (~top 3%)

Missense Constraint
1.82Z-score
OE missense 0.79 (0.740.86)
495 obs / 623.0 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.00 (0.000.09)
00.351.4
Missense OE0.79 (0.740.86)
00.61.4
Synonymous OE0.90
01.21.6
LoF obs/exp: 0 / 34.1Missense obs/exp: 495 / 623.0Syn Z: 1.30
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveNHS-related Nance-Horan syndromeLOFXLR
DN
0.2299th %ile
GOF
0.3689th %ile
LOF
0.86top 5%

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

LOFprediction above median · 52% of P/LP variants are LoF · LOEUF 0.09

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

500 submitted variants in ClinVar

Classification Summary

Pathogenic37
Likely Pathogenic17
VUS191
Likely Benign82
Benign26
Conflicting8
37
Pathogenic
17
Likely Pathogenic
191
VUS
82
Likely Benign
26
Benign
8
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
12
0
25
0
37
Likely Pathogenic
16
0
1
0
17
VUS
0
175
14
2
191
Likely Benign
1
19
13
49
82
Benign
0
6
10
10
26
Conflicting
8
Total292006361361

LoF = frameshift, stop gained/lost, canonical splice · Counts from ClinVar esearch · Updated hourly

View in ClinVar →

Protein Context — Lollipop Plot

NHS · 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

Fetal Developmental AbnormalityFetal DevelopmentHuman

Scottish Advanced Fetal Research Study

RECRUITING
NCT04613583University of AberdeenStarted 2016-05-01
SAFeR fetuses
X-Linked Retinitis Pigmentosa

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

ACTIVE NOT RECRUITING
NCT04794101Phase PHASE3Janssen Research & Development, LLCStarted 2020-12-04
Genetic: AAV5-hRKp.RPGR Intermediate DoseGenetic: AAV5-hRKp.RPGR Low Dose
Hormone Refractory Prostate Cancer

The BARCODE 2 Study - The Use of Genetic Profiling to Guide Prostate Cancer Treatment

ACTIVE NOT RECRUITING
NCT02955082Phase PHASE2Institute of Cancer Research, United KingdomStarted 2017-05-25
Carboplatin
Peripheral Arterial DiseaseSilent Atherosclerosis

Mechanistic Study of Anti-Platelet Therapy in Atherosclerosis

NOT YET RECRUITING
NCT07169513Phase NAGuy's and St Thomas' NHS Foundation TrustStarted 2025-10-01
Aspirin 75 mg dailyClopidogrel 75 mg dailyClopidgrel 75 mg daily
Citrin Deficiency

Multi-omics Study in Citrin Deficiency

ACTIVE NOT RECRUITING
NCT06895746Johannes HaeberleStarted 2023-04-01
Rett Syndrome

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

ACTIVE NOT RECRUITING
NCT06152237Phase PHASE1, PHASE2Taysha Gene Therapies, Inc.Started 2023-12-12
TSHA-102
Advanced Solid Tumor

Study of RP3 Monotherapy and RP3 in Combination With Nivolumab in Patients With Solid Tumours

ACTIVE NOT RECRUITING
NCT04735978Phase PHASE1Replimune, Inc.Started 2020-12-29
RP3Nivolumab
Rett Syndrome

A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

RECRUITING
NCT05898620Phase PHASE3Neurogene Inc.Started 2023-06-13
NGN-401
Hemophilia B

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

RECRUITING
NCT06379789Phase PHASE1, PHASE2Regeneron PharmaceuticalsStarted 2024-09-11
REGV131LNP1265
ObesityType2diabetesLipodystrophy

Susceptibility to Infectious Diseases in obEsity: an endocRine trAnslational socioLogic Evaluation, "SIDERALE"

RECRUITING
NCT06236932Phase NAFederico II UniversityStarted 2023-12-29
Mediterranean dietMelatonin supplementation
Breast Cancer

Trial of Perioperative Endocrine Therapy - Individualising Care

ACTIVE NOT RECRUITING
NCT02338310Phase PHASE3Institute of Cancer Research, United KingdomStarted 2008-09
Aromatase Inhibitors
Diabetes MellitusMonogenic DiabetesHyperinsulinism

Understanding Beta Cell Disorders Through the Study of Rare Genotypes (ENDURE)

RECRUITING
NCT06478121University of ExeterStarted 2025-11-11
Data and Blood collectionMRI
Clinical Literature
Open Research Assistant →
Key Publications
Landmark & review papers · by relevance
PubMed
Top 5 results · since 2015Search PubMed ↗