PEX1

Chr 7AR

peroxisomal biogenesis factor 1

Also known as: HMLR1, PBD1A, PBD1B, ZWS, ZWS1

This gene encodes a member of the AAA ATPase family, a large group of ATPases associated with diverse cellular activities. This protein is cytoplasmic but is often anchored to a peroxisomal membrane where it forms a heteromeric complex and plays a role in the import of proteins into peroxisomes and peroxisome biogenesis. Mutations in this gene have been associated with complementation group 1 peroxisomal disorders such as neonatal adrenoleukodystrophy, infantile Refsum disease, and Zellweger syndrome. Alternatively spliced transcript variants have been found for this gene. [provided by RefSeq, Sep 2013]

GeneReviewsOMIMResearchGenerating clinical summary…

Primary Disease Associations & Inheritance

Heimler syndrome 1MIM #234580
AR
Peroxisome biogenesis disorder 1A (Zellweger)MIM #214100
AR
Peroxisome biogenesis disorder 1B (NALD/IRD)MIM #601539
AR

Clinical highlights

Gene-disease validity (ClinGen)
peroxisome biogenesis disorder due to PEX1 defect · ARDefinitivesufficient evidence for diagnostic panels
Interpreting a novel variant
Loss of function is the curated mechanism (Gene2Phenotype), though the gene is not strongly LoF-constrained in the population — weigh truncating variants against that tolerance.Curated gene-level mechanism — a prior for triage, not a per-variant call.
3
Active trials
28
Pubs (1 yr)
P/LP submissions
P/LP missense
0.66
LOEUF
LOF
Mechanism· G2P
📖
GeneReview available — PEX1
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?
0.66LOEUF
pLI 0.000
Z-score 3.86
OE 0.50 (0.380.66)
Tolerant

Typical tolerance to LoF variation

Missense Constraint?
1.14Z-score
OE missense 0.88 (0.820.94)
582 obs / 664.4 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios?
LoF OE?0.50 (0.380.66)
00.351.4
Missense OE?0.88 (0.820.94)
00.61.4
Synonymous OE?0.92
01.21.6
LoF obs/exp: 35 / 69.8Missense obs/exp: 582 / 664.4Syn Z: 0.96

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

PEX1 · protein map & ClinVar variants

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