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GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 15 days ago
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AOPEP
aminopeptidase O (putative)
Chromosome 9 Β· 9q22.32
NCBI Gene: 84909Ensembl: ENSG00000148120.20HGNC: HGNC:1361UniProt: Q8N6M6
26PubMed Papers
21Diseases
1Drugs
9Pathogenic Variants
FUNCTIONAL ROLE
Protease
CLINICAL
Clinical TrialsOMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
nucleolusmetallopeptidase activityzinc ion bindingmetalloaminopeptidase activitydystonia 31atrial fibrillationheart failurepolycystic ovary syndrome
✦AI Summary

AOPEP encodes aminopeptidase O, a zinc-dependent proteolytic processing enzyme that catalyzes hydrolysis of amino acid residues from the N-terminus of peptide and protein substrates 1. The protein is preferentially expressed in glial cells and is potentially linked to endosomal-lysosomal pathways and synaptogenesis 12. AOPEP is associated with autosomal recessive dystonia (Zech-Boesch syndrome), with 74% of disease-associated alleles being protein-truncating variants including stop-gain, frameshift, and splice-site mutations 1. Affected individuals present with childhood to adult-onset dystonia, frequently generalizing across body regions (60% of cases), and variable expressivity suggesting age-dependent penetrance 13. Biallelic loss-of-function variants cause progressive dystonia with prominent limb involvement, occasionally accompanied by parkinsonism 24. Functionally, AOPEP variants show convergent neural dynamics in the globus pallidus characterized by altered firing regularity and bursting patterns comparable to other dystonia genes, despite lacking shared molecular pathways 5. Additionally, AOPEP promoter SNPs regulate alternative splicing affecting miR-27b-3p expression, relevant to kidney fibrosis in diabetic nephropathy 6. AOPEP genetic variants also show evidence of positive selection in polycystic ovary syndrome susceptibility 7.

Sources cited
1
AOPEP encodes aminopeptidase O, a proteolytic processing enzyme preferentially expressed in glia and linked to endosomal-lysosomal pathways; most AOPEP variants are protein-truncating; causes autosomal recessive dystonia with variable onset and generalization
PMID: 40147878
2
Biallelic AOPEP loss-of-function variants cause progressive dystonia affecting limbs; AOPEP is a zinc-dependent aminopeptidase involved in synaptogenesis and neural maintenance
PMID: 34596301
3
AOPEP variants enriched in dystonia cohorts; presents with childhood to adult-onset dystonia with variable phenotypes from isolated cervical to segmental dystonia
PMID: 36933031
4
Biallelic AOPEP loss-of-function variants cause adolescence-onset generalized dystonia and dystonia-parkinsonism in diverse ethnic populations
PMID: 35306330
5
AOPEP dystonia shows distinct pallidal neural dynamics with altered firing regularity compared to other genetic dystonia genes
PMID: 39887724
6
AOPEP promoter SNPs regulate alternative splicing affecting miR-27b-3p expression in kidney disease context
PMID: 40934963
7
AOPEP variants show evidence of positive selection in polycystic ovary syndrome susceptibility
PMID: 37982420
Disease Associationsβ“˜21
dystonia 31Open Targets
0.65Moderate
atrial fibrillationOpen Targets
0.54Moderate
heart failureOpen Targets
0.47Moderate
polycystic ovary syndromeOpen Targets
0.47Moderate
cardiac arrhythmiaOpen Targets
0.44Moderate
atrial flutterOpen Targets
0.43Moderate
SnoringOpen Targets
0.41Moderate
nephrolithiasisOpen Targets
0.38Weak
Alzheimer diseaseOpen Targets
0.33Weak
Hodgkins lymphomaOpen Targets
0.33Weak
Abnormality of the skeletal systemOpen Targets
0.32Weak
anovulationOpen Targets
0.31Weak
female infertilityOpen Targets
0.31Weak
ovarian dysfunctionOpen Targets
0.30Weak
musculoskeletal system diseaseOpen Targets
0.30Weak
leukoplakia of penisOpen Targets
0.30Weak
Back painOpen Targets
0.29Weak
portal hypertensionOpen Targets
0.29Weak
cardioembolic strokeOpen Targets
0.28Weak
chronic obstructive pulmonary diseaseOpen Targets
0.27Weak
Dystonia 31UniProt
Pathogenic Variants9
NM_001193329.3(AOPEP):c.703C>T (p.Gln235Ter)Likely pathogenic
Dystonia 31
β˜…β˜…β˜†β˜†2023β†’ Residue 235
NM_001193329.3(AOPEP):c.763C>T (p.Arg255Ter)Likely pathogenic
Dystonia 31|Clear cell carcinoma of kidney|Sarcoma
β˜…β˜†β˜†β˜†2023β†’ Residue 255
NM_001193329.3(AOPEP):c.219del (p.Lys73fs)Likely pathogenic
Dystonia 31
β˜…β˜†β˜†β˜†2023β†’ Residue 73
NM_001193329.3(AOPEP):c.1215del (p.Val406fs)Likely pathogenic
Dystonia 31|AOPEP-related disorder
β˜…β˜†β˜†β˜†2023β†’ Residue 406
NM_001193329.3(AOPEP):c.2207dup (p.Tyr736Ter)Likely pathogenic
Dystonia 31
β˜…β˜†β˜†β˜†2023β†’ Residue 736
NM_001193329.3(AOPEP):c.1477C>T (p.Arg493Ter)Pathogenic
Dystonia 31
β˜…β˜†β˜†β˜†β†’ Residue 493
NM_001193329.3(AOPEP):c.1744del (p.Met582fs)Pathogenic
Dystonia 31
β˜…β˜†β˜†β˜†β†’ Residue 582
NM_001193329.3(AOPEP):c.964+2T>GPathogenic
Dystonia 31
β˜…β˜†β˜†β˜†
NM_001193329.3(AOPEP):c.777G>A (p.Trp259Ter)Pathogenic
Dystonia 31
β˜†β˜†β˜†β˜†2022β†’ Residue 259
View on ClinVar β†—
Drug Targets1
TOSEDOSTATPhase II
Aminopeptidase inhibitor
acute myeloid leukemia by FAB classification
Related Genes
DENND1AProtein interaction74%
Tissue Expression6 tissues
Liver
100%
Heart
98%
Ovary
41%
Lung
29%
Brain
14%
Bone Marrow
5%
Gene Interaction Network
Click a node to explore
AOPEPDENND1A
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted Β· UniProt Q8N6M6
View on AlphaFold β†—
Constraintβ“˜
LOEUFβ“˜
0.98LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.78 [0.63–0.98]
RankingsWhere AOPEP stands among ~20K protein-coding genes
  • #12,732of 20,598
    Most Researched26
  • #2,993of 5,498
    Most Pathogenic Variants9
  • #9,333of 17,882
    Most Constrained (LOEUF)0.98
Genes detectedAOPEP
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
Evidence of positive selection of genetic variants associated with PCOS.
PMID: 37982420
Hum Reprod Β· 2023
1.00
2
PMID: 40147878
J Med Genet Β· 2025
0.90
3
Genome sequencing reanalysis increases the diagnostic yield in dystonia.
PMID: 38772265
Parkinsonism Relat Disord Β· 2024
0.80
4
A renal biopsy-anchored multi-marker signature involving AOPEP SNP-driven splicing, miR-27b-3p and glycated albumin for stratifying renal damage in type 2 diabetes.
PMID: 40934963
Diabetes Res Clin Pract Β· 2025
0.70
5
Spiking Patterns in the Globus Pallidus Highlight Convergent Neural Dynamics across Diverse Genetic Dystonia Syndromes.
PMID: 39887724
Ann Neurol Β· 2025
0.60