HomeAboutRankingsData Sources
Β© 2026 GeneE
🧬
GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
β“˜GeneE is for informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment.
PEX1
peroxisomal biogenesis factor 1
Chromosome 7 Β· 7q21.2
NCBI Gene: 5189Ensembl: ENSG00000127980.16HGNC: HGNC:8850UniProt: A0A0C4DG33
97PubMed Papers
24Diseases
0Drugs
469Pathogenic Variants
FUNCTIONAL ROLE
Transporter
RESEARCH IMPACT
Variant-Rich
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
microtubule-based peroxisome localizationprotein bindingATP bindingATP hydrolysis activityZellweger syndromeperoxisome biogenesis disorder 1Bperoxisome biogenesis disorder 1A (Zellweger)peroxisome biogenesis disorder
✦AI Summary

PEX1 encodes a 143-kDa AAA-ATPase that forms a heterohexameric complex with PEX6, essential for peroxisome biogenesis and maintenance 1. The PEX1-PEX6 complex functions as a protein dislocase that extracts monoubiquitinated PEX5 receptor from peroxisomal membranes through the PEX2-PEX10-PEX12 retrotranslocation channel via ATP-dependent mechanical unfolding, enabling PEX5 recycling and peroxisomal matrix protein import 2. This extraction process unfolds PEX5's TPR repeats and releases bound cargo 1. Mutations in PEX1 are the most common cause of peroxisome biogenesis disorders (PBDs), accounting for approximately 65% of cases 3. Disease severity correlates with mutation type: truncating mutations on both alleles cause severe Zellweger syndrome, while missense mutations typically produce milder phenotypes like neonatal adrenoleukodystrophy and infantile Refsum disease 4. Heimler syndrome, caused by biallelic PEX1 mutations including the common G843D variant, represents a less severe form presenting with sensorineural hearing loss, retinal dystrophy, and amelogenesis imperfecta 5. The G843D mutation reduces PEX1 stability and PEX6 binding affinity, leading to proteasomal degradation; protein stabilization strategies show therapeutic potential 2.

Sources cited
1
PEX1 and PEX6 form a heterohexameric AAA-ATPase capable of unfolding substrates; mutations cause peroxisome biogenesis disorders including Zellweger syndrome
PMID: 35805150
2
PEX1-PEX6 complex required for peroxisomal matrix protein import; G843D mutation reduces PEX1 stability and PEX6 binding; protein stabilization is therapeutic strategy
PMID: 40158855
3
Approximately 65% of peroxisome biogenesis disorder patients have mutations in PEX1/PEX6; missense mutations have different functional impacts than fold-destabilizing mutations
PMID: 31374812
4
PEX1 mutations are the most common cause of Zellweger spectrum diseases; disease severity correlates with mutation type (truncating vs. missense)
PMID: 16086329
5
Heimler syndrome is a rare, less severe form of peroxisome biogenesis disorders caused by PEX1 or PEX6 biallelic mutations, presenting with sensorineural hearing loss, retinal dystrophy, and amelogenesis imperfecta
PMID: 33417209
Disease Associationsβ“˜24
Zellweger syndromeOpen Targets
0.83Strong
peroxisome biogenesis disorder 1BOpen Targets
0.82Strong
peroxisome biogenesis disorder 1A (Zellweger)Open Targets
0.81Strong
peroxisome biogenesis disorderOpen Targets
0.81Strong
peroxisome biogenesis disorder due to PEX1 defectOpen Targets
0.67Moderate
Zellweger spectrum disordersOpen Targets
0.65Moderate
Peroxisome biogenesis disorder-Zellweger syndrome spectrumOpen Targets
0.62Moderate
Retinal dystrophyOpen Targets
0.53Moderate
genetic disorderOpen Targets
0.52Moderate
nervous system benign neoplasmOpen Targets
0.43Moderate
optic atrophyOpen Targets
0.38Weak
amelogenesis imperfectaOpen Targets
0.37Weak
peroxisome biogenesis disorder 2BOpen Targets
0.37Weak
peroxisome biogenesis disorder type 3BOpen Targets
0.37Weak
peroxisomal diseaseOpen Targets
0.34Weak
Abnormality of metabolism/homeostasisOpen Targets
0.34Weak
neurodegenerative diseaseOpen Targets
0.33Weak
hypopituitarismOpen Targets
0.26Weak
X-linked adrenoleukodystrophyOpen Targets
0.19Weak
adrenoleukodystrophyOpen Targets
0.19Weak
Heimler syndrome 1UniProt
Peroxisome biogenesis disorder 1AUniProt
Peroxisome biogenesis disorder 1BUniProt
Peroxisome biogenesis disorder complementation group 1UniProt
Pathogenic Variants469
NM_000466.3(PEX1):c.1099del (p.Gln367fs)Pathogenic
not provided|Heimler syndrome 1|Zellweger spectrum disorders|Peroxisome biogenesis disorder due to PEX1 defect|Peroxisome biogenesis disorder 1A (Zellweger)
β˜…β˜…β˜†β˜†2026β†’ Residue 367
NM_000466.3(PEX1):c.2916del (p.Gly973fs)Pathogenic
Peroxisome biogenesis disorder 1A (Zellweger)|not provided|Peroxisome biogenesis disorder|Peroxisome biogenesis disorder 1A (Zellweger);Peroxisome biogenesis disorder 1B|Zellweger spectrum disorders|Peroxisome biogenesis disorder due to PEX1 defect|Heimler syndrome 1|Peroxisome biogenesis disorder 1A (Zellweger);Heimler syndrome 1;Peroxisome biogenesis disorder 1B
β˜…β˜…β˜†β˜†2026β†’ Residue 973
NM_000466.3(PEX1):c.2992C>T (p.Arg998Ter)Pathogenic
Peroxisome biogenesis disorder|Peroxisome biogenesis disorder 1A (Zellweger)|Heimler syndrome 1|Peroxisome biogenesis disorder 1B|not provided|Zellweger spectrum disorders|Inborn genetic diseases
β˜…β˜…β˜†β˜†2026β†’ Residue 998
NM_000466.3(PEX1):c.2097dup (p.Ile700fs)Pathogenic
Peroxisome biogenesis disorder 1A (Zellweger)|not provided|Heimler syndrome 1|Peroxisome biogenesis disorder|Peroxisome biogenesis disorder 1B;Peroxisome biogenesis disorder 1A (Zellweger);Heimler syndrome 1|Peroxisome biogenesis disorder 1B|Peroxisome biogenesis disorder 1B;Peroxisome biogenesis disorder 1A (Zellweger)|Retinal dystrophy|Zellweger spectrum disorders|Inborn genetic diseases|PEX1-related disorder|Optic atrophy
β˜…β˜…β˜†β˜†2026β†’ Residue 700
NM_000466.3(PEX1):c.2966T>C (p.Ile989Thr)Pathogenic
Heimler syndrome 1|Peroxisome biogenesis disorder 1B;Peroxisome biogenesis disorder 1A (Zellweger)|Zellweger spectrum disorders|not provided|Inborn genetic diseases|Peroxisome biogenesis disorder|Peroxisome biogenesis disorder 1B;Peroxisome biogenesis disorder 1A (Zellweger);Heimler syndrome 1
β˜…β˜…β˜†β˜†2026β†’ Residue 989
NM_000466.3(PEX1):c.2528G>A (p.Gly843Asp)Pathogenic
Peroxisome biogenesis disorder|Leber congenital amaurosis|Peroxisome biogenesis disorder 1A (Zellweger)|not provided|Peroxisome biogenesis disorder 1B|Heimler syndrome 1|Peroxisome biogenesis disorder 1A (Zellweger);Peroxisome biogenesis disorder 1B;Heimler syndrome 1|Peroxisomal disorder|Peroxisome biogenesis disorder 1A (Zellweger);Peroxisome biogenesis disorder 1B|Retinal dystrophy|Inborn genetic diseases|Zellweger spectrum disorders|not specified|PEX1-related disorder|Peroxisome biogenesis disorder due to PEX1 defect
β˜…β˜…β˜†β˜†2026β†’ Residue 843
NM_000466.3(PEX1):c.2875C>T (p.Arg959Ter)Pathogenic
Peroxisome biogenesis disorder 1A (Zellweger)|Peroxisome biogenesis disorder 1B|Zellweger spectrum disorders|Peroxisome biogenesis disorder 1A (Zellweger);Peroxisome biogenesis disorder 1B|not provided|Heimler syndrome 1
β˜…β˜…β˜†β˜†2026β†’ Residue 959
NM_000466.3(PEX1):c.2926+1G>APathogenic
not provided|Peroxisome biogenesis disorder|Peroxisome biogenesis disorder 1B|Peroxisome biogenesis disorder 1A (Zellweger);Peroxisome biogenesis disorder 1B|Zellweger spectrum disorders|Inborn genetic diseases|Heimler syndrome 1|Peroxisome biogenesis disorder 1A (Zellweger)
β˜…β˜…β˜†β˜†2026
NM_000466.3(PEX1):c.2420T>A (p.Leu807Ter)Pathogenic
Zellweger spectrum disorders
β˜…β˜…β˜†β˜†2026β†’ Residue 807
NM_000466.3(PEX1):c.2859dup (p.Thr954fs)Pathogenic
Peroxisome biogenesis disorder 1A (Zellweger)|Peroxisome biogenesis disorder 1B|Zellweger spectrum disorders|Peroxisome biogenesis disorder 1A (Zellweger);Heimler syndrome 1;Peroxisome biogenesis disorder 1B|Heimler syndrome 1
β˜…β˜…β˜†β˜†2026β†’ Residue 954
NM_000466.3(PEX1):c.2368C>T (p.Arg790Ter)Pathogenic
Peroxisome biogenesis disorder|Zellweger spectrum disorders|Heimler syndrome 1|not provided
β˜…β˜…β˜†β˜†2026β†’ Residue 790
NM_000466.3(PEX1):c.2226+2T>CPathogenic
not provided|Zellweger spectrum disorders
β˜…β˜…β˜†β˜†2026
NM_000466.3(PEX1):c.2845C>T (p.Arg949Trp)Pathogenic
Zellweger spectrum disorders|Peroxisome biogenesis disorder 1A (Zellweger)|Heimler syndrome 1|Peroxisome biogenesis disorder 1A (Zellweger);Heimler syndrome 1;Peroxisome biogenesis disorder 1B
β˜…β˜…β˜†β˜†2025β†’ Residue 949
NM_000466.3(PEX1):c.2383C>T (p.Arg795Ter)Pathogenic
Peroxisome biogenesis disorder 1A (Zellweger)|not provided|Peroxisome biogenesis disorder 1A (Zellweger);Peroxisome biogenesis disorder 1B|Zellweger spectrum disorders|Peroxisome biogenesis disorder|Heimler syndrome 1|Peroxisome biogenesis disorder 1A (Zellweger);Heimler syndrome 1;Peroxisome biogenesis disorder 1B
β˜…β˜…β˜†β˜†2025β†’ Residue 795
NM_000466.3(PEX1):c.2T>G (p.Met1Arg)Pathogenic
Peroxisome biogenesis disorder 1A (Zellweger)|Peroxisome biogenesis disorder 1B|Zellweger spectrum disorders|Peroxisome biogenesis disorder 1A (Zellweger);Heimler syndrome 1;Peroxisome biogenesis disorder 1B
β˜…β˜…β˜†β˜†2025β†’ Residue 1
NM_000466.3(PEX1):c.1952_1960dup (p.Trp653_Met654insThrValTrp)Pathogenic
not provided|Peroxisome biogenesis disorder|Peroxisome biogenesis disorder 1A (Zellweger)|Zellweger spectrum disorders|Peroxisome biogenesis disorder 1B;Peroxisome biogenesis disorder 1A (Zellweger)|Heimler syndrome 1
β˜…β˜…β˜†β˜†2025β†’ Residue 653
NM_000466.3(PEX1):c.2T>C (p.Met1Thr)Pathogenic
Peroxisome biogenesis disorder 1B|Peroxisome biogenesis disorder 1A (Zellweger)|Peroxisome biogenesis disorder type 1A|Peroxisome biogenesis disorder|Zellweger spectrum disorders|Peroxisome biogenesis disorder 1A (Zellweger);Heimler syndrome 1;Peroxisome biogenesis disorder 1B
β˜…β˜…β˜†β˜†2025β†’ Residue 1
NM_000466.3(PEX1):c.3180dup (p.Gly1061fs)Pathogenic
Peroxisome biogenesis disorder|Zellweger spectrum disorders|Heimler syndrome 1
β˜…β˜…β˜†β˜†2025β†’ Residue 1061
NM_000466.3(PEX1):c.2846G>A (p.Arg949Gln)Pathogenic
Peroxisome biogenesis disorder 1A (Zellweger)|Zellweger spectrum disorders|not provided|Heimler syndrome 1
β˜…β˜…β˜†β˜†2025β†’ Residue 949
NM_000466.3(PEX1):c.829C>T (p.Gln277Ter)Pathogenic
not provided|Zellweger spectrum disorders
β˜…β˜…β˜†β˜†2025β†’ Residue 277
View on ClinVar β†—
Related Genes
PEX13Protein interaction98%ABCD3Protein interaction94%PEX5LProtein interaction90%PEX6Protein interaction81%ZFAND6Protein interaction77%PEX12Protein interaction75%
Tissue Expression6 tissues
Heart
100%
Ovary
88%
Bone Marrow
72%
Brain
67%
Liver
64%
Lung
53%
Gene Interaction Network
Click a node to explore
PEX1PEX13ABCD3PEX5LPEX6ZFAND6PEX12
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted Β· UniProt O43933
View on AlphaFold β†—
Constraintβ“˜
LOEUFβ“˜
0.75LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.60 [0.49–0.75]
RankingsWhere PEX1 stands among ~20K protein-coding genes
  • #4,957of 20,598
    Most Researched97 Β· top quartile
  • #115of 5,498
    Most Pathogenic Variants469 Β· top 5%
  • #5,886of 17,882
    Most Constrained (LOEUF)0.75
Genes detectedPEX1
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
PMID: 20301621
1.00
2
Heimler Syndrome.
PMID: 33417209
Adv Exp Med Biol Β· 2020
0.90
3
Insights into the Structure and Function of the Pex1/Pex6 AAA-ATPase in Peroxisome Homeostasis.
PMID: 35805150
Cells Β· 2022
0.80
4
PEX1
PMID: 40158855
J Biol Chem Β· 2025
0.70
5
Structural Mapping of Missense Mutations in the Pex1/Pex6 Complex.
PMID: 31374812
Int J Mol Sci Β· 2019
0.60