NPHP3

Chr 3AR

nephrocystin 3

Also known as: CFAP31, MKS7, NPH3, RHPD, RHPD1, SLSN3

The protein is required for normal ciliary development and function, regulates Wnt signaling pathways, and is essential for renal tubular development and convergent extension cell movements. Mutations cause autosomal recessive ciliopathies including nephronophthisis type 3 (progressive kidney disease), Meckel syndrome type 7 (severe developmental disorder), and renal-hepatic-pancreatic dysplasia affecting multiple organ systems. The gene is highly constrained against loss-of-function variants (LOEUF 0.649), indicating intolerance to protein-disrupting mutations.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt
LOFmechanismARLOEUF 0.654 OMIM phenotypes
Clinical SummaryNPHP3
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Gene-Disease Validity (ClinGen)
nephronophthisis · ARDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
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ClinVar Variants
72 unique Pathogenic / Likely Pathogenic· 241 VUS of 500 total submissions
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GeneReview available — NPHP3
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.65LOEUF
pLI 0.000
Z-score 4.10
OE 0.50 (0.380.65)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
0.86Z-score
OE missense 0.91 (0.850.97)
627 obs / 690.9 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.50 (0.380.65)
00.351.4
Missense OE0.91 (0.850.97)
00.61.4
Synonymous OE0.89
01.21.6
LoF obs/exp: 38 / 76.7Missense obs/exp: 627 / 690.9Syn Z: 1.41
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveNPHP3-related Senior-Loken syndromeOTHERAR
definitiveNPHP3-related nephronophthisisLOFAR

Predictions shown for reference only — model trained on dominant genes, not applicable to AR conditions.

DN
0.6161th %ile
GOF
0.6247th %ile
LOF
0.3356th %ile

The Badonyi & Marsh prediction model was trained exclusively on dominant disease genes. Predictions are not reliable for genes with autosomal recessive inheritance and are shown at reduced opacity for reference only.

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

ClinVar Variant Classifications

500 submitted variants in ClinVar

Classification Summary

Pathogenic38
Likely Pathogenic34
VUS241
Likely Benign176
Benign4
Conflicting3
38
Pathogenic
34
Likely Pathogenic
241
VUS
176
Likely Benign
4
Benign
3
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
33
0
5
0
38
Likely Pathogenic
30
2
2
0
34
VUS
1
216
19
5
241
Likely Benign
0
1
88
87
176
Benign
0
0
3
1
4
Conflicting
3
Total6421911793496

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

View in ClinVar →

Protein Context — Lollipop Plot

NPHP3 · 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 →