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GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
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AMPD2
adenosine monophosphate deaminase 2
Chromosome 1 Β· 1p13.3
NCBI Gene: 271Ensembl: ENSG00000116337.21HGNC: HGNC:469UniProt: Q01433
74PubMed Papers
22Diseases
0Drugs
49Pathogenic Variants
FUNCTIONAL ROLE
Hub Gene
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
IMP biosynthetic processAMP deaminase activityprotein bindingcyclic purine nucleotide metabolic processpontocerebellar hypoplasia type 9pontocerebellar hypoplasiahereditary spastic paraplegia 63hereditary spastic paraplegia
✦AI Summary

AMPD2 (adenosine monophosphate deaminase 2) is a key metabolic enzyme that catalyzes the deamination of AMP to IMP, playing a critical role in the purine nucleotide cycle and energy homeostasis 1. The gene is widely expressed in non-muscle tissues and encodes isoform L, the predominant AMP deaminase activity in adult human liver 2. AMPD2 is located on chromosome 1.3 and contains 19 exons with alternative transcription patterns that generate tissue-specific isoforms with variable N-terminal extensions 32. AMPD2 deficiency causes selective neurodegeneration, particularly in the hippocampal dentate gyrus, through disrupted purine metabolism and reduced GTP synthesis; IMPDH2 filament formation in resistant regions protects against neuronal vulnerability 4. AMPD2 dysfunction also impairs renal function, with deficiency causing proteinuria and podocyte dysfunction resembling minimal-change nephropathy 5. In hereditary fructose intolerance, AMPD2 activation drives metabolic dysregulation and liver disease, and its hepatic deletion improves metabolic consequences 6. Recent evidence suggests AMPD2 as a biomarker for acute myocardial infarction diagnosis through lactylation-related pathways 7. Mutations cause childhood neurodegenerative disorders including pontocerebellar hypoplasia 9 and spastic paraplegia 63, underscoring its essential role in neural energy metabolism.

Sources cited
1
AMPD2 encodes isoform L, catalyzes AMP deamination to IMP, plays role in purine nucleotide cycle
PMID: 1429593
2
AMPD2 is widely expressed in non-muscle tissues and generates alternative transcripts with variable N-terminal extensions
PMID: 8526848
3
AMPD2 gene located on chromosome 1p13.3, contains 19 exons, regulated by two alternative promoters
PMID: 8764830
4
AMPD2 deficiency causes selective hippocampal dentate gyrus neurodegeneration; IMPDH2 filaments protect vulnerable regions
PMID: 39075237
5
AMPD2 deficiency causes proteinuria and podocyte dysfunction in kidneys
PMID: 22212473
6
AMPD2 activation drives metabolic dysregulation in hereditary fructose intolerance; deletion improves metabolic consequences
PMID: 38992061
7
AMPD2 identified as lactylation-related gene with potential as acute myocardial infarction biomarker
PMID: 39521235
Disease Associationsβ“˜22
pontocerebellar hypoplasia type 9Open Targets
0.81Strong
pontocerebellar hypoplasiaOpen Targets
0.66Moderate
hereditary spastic paraplegia 63Open Targets
0.65Moderate
hereditary spastic paraplegiaOpen Targets
0.60Moderate
Non-syndromic pontocerebellar hypoplasiaOpen Targets
0.48Moderate
Autosomal recessive spastic paraplegia type 63Open Targets
0.46Moderate
genetic disorderOpen Targets
0.38Weak
Abnormality of the nervous systemOpen Targets
0.34Weak
spastic ataxiaOpen Targets
0.34Weak
Global developmental delayOpen Targets
0.26Weak
cerebral malformationOpen Targets
0.26Weak
SeizureOpen Targets
0.26Weak
microcephalyOpen Targets
0.11Weak
Intellectual disabilityOpen Targets
0.11Weak
colorectal carcinomaOpen Targets
0.08Suggestive
nutritional deficiency diseaseOpen Targets
0.08Suggestive
nephrotic syndromeOpen Targets
0.08Suggestive
focal segmental glomerulosclerosisOpen Targets
0.07Suggestive
Blackfan-Diamond anemiaOpen Targets
0.07Suggestive
hypertriglyceridemia 2Open Targets
0.07Suggestive
Pontocerebellar hypoplasia 9UniProt
Spastic paraplegia 63, autosomal recessiveUniProt
Pathogenic Variants49
NM_001368809.2(AMPD2):c.1057C>T (p.Arg353Ter)Pathogenic
Pontocerebellar hypoplasia type 9|Pontocerebellar hypoplasia type 9;Hereditary spastic paraplegia 63|not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 353
NM_001368809.2(AMPD2):c.102_103del (p.Gly35fs)Pathogenic
not provided|Hereditary spastic paraplegia 63;Pontocerebellar hypoplasia type 9
β˜…β˜…β˜†β˜†2025β†’ Residue 35
NM_001368809.2(AMPD2):c.1698+1G>APathogenic
Hereditary spastic paraplegia 63;Pontocerebellar hypoplasia type 9|Inborn genetic diseases
β˜…β˜…β˜†β˜†2025
NM_001368809.2(AMPD2):c.1859G>A (p.Arg620His)Pathogenic
Pontocerebellar hypoplasia type 9|Pontocerebellar hypoplasia type 9;Hereditary spastic paraplegia 63|Hereditary spastic paraplegia 63|Pontoneocerebellar hypoplasia
β˜…β˜…β˜†β˜†2025β†’ Residue 620
NM_001368809.2(AMPD2):c.520G>T (p.Glu174Ter)Pathogenic
not provided|Pontocerebellar hypoplasia type 9|Pontoneocerebellar hypoplasia|Hereditary spastic paraplegia 63;Pontocerebellar hypoplasia type 9
β˜…β˜…β˜†β˜†2025β†’ Residue 174
NM_001368809.2(AMPD2):c.252_253insAGTGAGC (p.Glu85fs)Likely pathogenic
Pontocerebellar hypoplasia type 9|not provided
β˜…β˜…β˜†β˜†2024β†’ Residue 85
NM_001368809.2(AMPD2):c.646del (p.Leu216fs)Pathogenic
Pontoneocerebellar hypoplasia|Hereditary spastic paraplegia 63;Pontocerebellar hypoplasia type 9
β˜…β˜…β˜†β˜†2024β†’ Residue 216
NM_001368809.2(AMPD2):c.1345C>T (p.Arg449Ter)Pathogenic
Pontocerebellar hypoplasia type 9|not provided|Pontoneocerebellar hypoplasia
β˜…β˜…β˜†β˜†2024β†’ Residue 449
NM_001368809.2(AMPD2):c.2172G>C (p.Glu724Asp)Pathogenic
Pontocerebellar hypoplasia type 9|Pontoneocerebellar hypoplasia
β˜…β˜†β˜†β˜†2026β†’ Residue 724
NM_001368809.2(AMPD2):c.-90_-69dupPathogenic
Pontocerebellar hypoplasia type 9;Hereditary spastic paraplegia 63
β˜…β˜†β˜†β˜†2026
NM_001368809.2(AMPD2):c.886_887del (p.Tyr295_Pro296insTer)Pathogenic
Hereditary spastic paraplegia 63;Pontocerebellar hypoplasia type 9
β˜…β˜†β˜†β˜†2026β†’ Residue 295
NM_001368809.2(AMPD2):c.2007C>A (p.Tyr669Ter)Pathogenic
Pontocerebellar hypoplasia type 9;Hereditary spastic paraplegia 63
β˜…β˜†β˜†β˜†2025β†’ Residue 669
NM_001368809.2(AMPD2):c.-42dupPathogenic
Pontocerebellar hypoplasia type 9;Hereditary spastic paraplegia 63
β˜…β˜†β˜†β˜†2025
NM_001368809.2(AMPD2):c.1080+2T>GLikely pathogenic
Pontocerebellar hypoplasia type 9;Hereditary spastic paraplegia 63
β˜…β˜†β˜†β˜†2025
NM_001368809.2(AMPD2):c.861-2A>GLikely pathogenic
Hereditary spastic paraplegia 63;Pontocerebellar hypoplasia type 9
β˜…β˜†β˜†β˜†2025
NM_001368809.2(AMPD2):c.2185G>C (p.Ala729Pro)Likely pathogenic
Pontocerebellar hypoplasia type 9
β˜…β˜†β˜†β˜†2024β†’ Residue 729
NM_001368809.2(AMPD2):c.42del (p.Lys14fs)Pathogenic
Pontocerebellar hypoplasia type 9;Hereditary spastic paraplegia 63
β˜…β˜†β˜†β˜†2024β†’ Residue 14
NM_001368809.2(AMPD2):c.1471G>A (p.Gly491Arg)Likely pathogenic
Hereditary spastic paraplegia 63|Gastric cancer
β˜…β˜†β˜†β˜†2024β†’ Residue 491
NM_001368809.2(AMPD2):c.1325del (p.Pro442fs)Likely pathogenic
Pontocerebellar hypoplasia type 9;Hereditary spastic paraplegia 63
β˜…β˜†β˜†β˜†2024β†’ Residue 442
NM_001368809.2(AMPD2):c.1984-2A>GLikely pathogenic
Hereditary spastic paraplegia 63;Pontocerebellar hypoplasia type 9
β˜…β˜†β˜†β˜†2024
View on ClinVar β†—
Related Genes
NT5C2Protein interaction97%NT5MProtein interaction96%PDE11AProtein interaction96%PDE4BProtein interaction95%PNPProtein interaction94%ATICProtein interaction93%
Tissue Expression6 tissues
Bone Marrow
100%
Liver
24%
Brain
22%
Ovary
19%
Lung
16%
Heart
5%
Gene Interaction Network
Click a node to explore
AMPD2NT5C2NT5MPDE11APDE4BPNPATIC
PROTEIN STRUCTURE
Preparing viewer…
PDB4NO3 Β· 1.70 Γ… Β· X-ray
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
0.70LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.56 [0.45–0.70]
RankingsWhere AMPD2 stands among ~20K protein-coding genes
  • #6,375of 20,598
    Most Researched74
  • #1,354of 5,498
    Most Pathogenic Variants49 Β· top quartile
  • #5,387of 17,882
    Most Constrained (LOEUF)0.70
Genes detectedAMPD2
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
Identification of a lactylation-related gene signature as the novel biomarkers for early diagnosis of acute myocardial infarction.
PMID: 39521235
Int J Biol Macromol Β· 2024
1.00
2
Cloning, sequence and characterization of the human AMPD2 gene: evidence for transcriptional regulation by two closely spaced promoters.
PMID: 8764830
Biochim Biophys Acta Β· 1996
0.90
3
Characterization of human AMP deaminase 2 (AMPD2) gene expression reveals alternative transcripts encoding variable N-terminal extensions of isoform L.
PMID: 8526848
Biochem J Β· 1995
0.80
4
IMPDH2 filaments protect from neurodegeneration in AMPD2 deficiency.
PMID: 39075237
EMBO Rep Β· 2024
0.70
5
Molecular cloning of AMP deaminase isoform L. Sequence and bacterial expression of human AMPD2 cDNA.
PMID: 1429593
J Biol Chem Β· 1992
0.60