PEX13

Chr 2AR

peroxisomal biogenesis factor 13

Also known as: NALD, PBD11A, PBD11B, ZWS

This gene encodes a peroxisomal membrane protein that binds the type 1 peroxisomal targeting signal receptor via a cytoplasmic SH3 domain, facilitating protein import into peroxisomes. Mutations cause autosomal recessive peroxisome biogenesis disorders including Zellweger syndrome (PBD11A) and the milder PBD11B phenotype. Loss-of-function mutations disrupt peroxisomal protein import and peroxisome assembly, leading to the characteristic metabolic and developmental abnormalities seen in these disorders.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
LOFmechanismARLOEUF 1.012 OMIM phenotypes
Clinical SummaryPEX13
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Gene-Disease Validity (ClinGen)
peroxisome biogenesis disorder · 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
40 unique Pathogenic / Likely Pathogenic· 175 VUS of 400 total submissions
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GeneReview available — PEX13
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
1.01LOEUF
pLI 0.000
Z-score 1.53
OE 0.58 (0.351.01)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint
-0.30Z-score
OE missense 1.06 (0.951.18)
230 obs / 217.7 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE0.58 (0.351.01)
00.351.4
Missense OE1.06 (0.951.18)
00.61.4
Synonymous OE1.08
01.21.6
LoF obs/exp: 9 / 15.5Missense obs/exp: 230 / 217.7Syn Z: -0.57
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitivePEX13-related peroxisome biogenesis disorder 11BLOFAR
definitivePEX13-related peroxisome biogenesis disorder 11A (Zellweger)LOFAR

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

DN
0.6937th %ile
GOF
0.6247th %ile
LOF
0.3355th %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

Pathogenic32
Likely Pathogenic8
VUS175
Likely Benign172
Benign1
32
Pathogenic
8
Likely Pathogenic
175
VUS
172
Likely Benign
1
Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
27
0
5
0
32
Likely Pathogenic
5
0
3
0
8
VUS
2
162
11
0
175
Likely Benign
0
0
42
130
172
Benign
0
0
1
0
1
Total3416262130388

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

View in ClinVar →

Protein Context — Lollipop Plot

PEX13 · 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 →
Full-Text Mentions
NLP-detected gene mentions in article bodies · via PubTator3
PubTator3
Top 4 full-text resultsSearch PubTator3 ↗