NPHS1

Chr 19AR

NPHS1 adhesion molecule, nephrin

Also known as: CNF, NPHN, nephrin

The protein is a transmembrane cell adhesion molecule that forms part of the slit diaphragm in glomerular podocytes, functioning as an ultrafilter to prevent albumin and other plasma proteins from entering urine. Mutations cause autosomal recessive congenital nephrotic syndrome type 1 (Finnish-type), characterized by severe proteinuria beginning in infancy with loss of normal kidney filtration barrier structure. This gene is extremely intolerant to loss-of-function variants, reflecting its critical role in kidney development and function.

Summary from RefSeq, OMIM, UniProt
Research Assistant →

Primary Disease Associations & Inheritance

Nephrotic syndrome, type 1MIM #256300
AR
Nephrotic syndrome, type 1MIM #256300
AR
3
Active trials
37
Pubs (1 yr)
65
P/LP submissions
4%
P/LP missense
0.70
LOEUF
LOF
Mechanism· G2P
Clinical SummaryNPHS1
🧬
Gene-Disease Validity (ClinGen)
congenital nephrotic syndrome, Finnish type · 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.
📋
ClinVar Variants
47 unique Pathogenic / Likely Pathogenic· 247 VUS of 400 total submissions
💊
Clinical Trials
3 active or recruiting trials — potential therapeutic options may be available
📖
GeneReview available — NPHS1
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
0.70LOEUF
pLI 0.000
Z-score 3.53
OE 0.52 (0.390.70)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
0.41Z-score
OE missense 0.96 (0.901.02)
698 obs / 729.0 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.52 (0.390.70)
00.351.4
Missense OE0.96 (0.901.02)
00.61.4
Synonymous OE1.07
01.21.6
LoF obs/exp: 33 / 63.4Missense obs/exp: 698 / 729.0Syn Z: -1.04
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveNPHS1-related nephrotic syndromeLOFAR

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

DN
0.7036th %ile
GOF
0.76top 25%
LOF
0.2970th %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

400 submitted variants in ClinVar

Classification Summary

Pathogenic12
Likely Pathogenic35
VUS247
Likely Benign81
Benign3
Conflicting21
12
Pathogenic
35
Likely Pathogenic
247
VUS
81
Likely Benign
3
Benign
21
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
6
0
6
0
12
Likely Pathogenic
26
2
7
0
35
VUS
0
195
38
14
247
Likely Benign
0
4
29
48
81
Benign
0
0
2
1
3
Conflicting
21
Total322018263399

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

View in ClinVar →

Protein Context — Lollipop Plot

NPHS1 · protein map & ClinVar variants

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

Clinical Literature
Open Research Assistant →
Full-Text Mentions
NLP-detected gene mentions in article bodies · via PubTator3
PubTator3
Top 5 full-text resultsSearch PubTator3 ↗