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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.
ABAT
4-aminobutyrate aminotransferase
Chromosome 16 Β· 16p13.2
NCBI Gene: 18Ensembl: ENSG00000183044.12HGNC: HGNC:23UniProt: H3BNQ7
58PubMed Papers
21Diseases
1Drugs
39Pathogenic Variants
CLINICAL
FDA Approved TargetOMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
4-aminobutyrate transaminase complexGABA metabolic processidentical protein bindingmitochondrionGamma-aminobutyric acid transaminase deficiencyGABA aminotransaminase deficiencyinfantile spasmsmitochondrial DNA depletion syndrome
✦AI Summary

ABAT (4-aminobutyrate aminotransferase) is a mitochondrial enzyme that catalyzes the conversion of gamma-aminobutyrate (GABA) to succinate semialdehyde, a critical step in GABA catabolism 1. The enzyme also converts L-beta-aminoisobutyrate to methylmalonate semialdehyde and functions as part of the mitochondrial 4-aminobutyrate:2-oxoglutarate transaminase complex, requiring pyridoxal phosphate as a cofactor 1. ABAT plays a crucial role in maintaining balanced mitochondrial nucleotide pools necessary for mitochondrial DNA synthesis and maintenance 2. Pathogenic variants in ABAT cause GABA-transaminase deficiency, leading to GABA accumulation and neurological dysfunction 1. This deficiency is associated with developmental and epileptic encephalopathy, complex partial epilepsy, developmental disability, cardiomyopathy, and blindness 1. The enzyme's role in GABA metabolism is fundamental to maintaining the balance between neuronal excitation and inhibition in the brain; mutations disrupt this balance and result in seizures and cognitive impairment 1. ABAT deficiency represents one of several genetic abnormalities affecting GABA metabolism that contribute to epilepsy pathogenesis, making it clinically significant for accurate diagnosis and treatment of metabolic forms of epilepsy.

Sources cited
1
ABAT catalyzes GABA conversion to succinate semialdehyde; mutations cause GABA-transaminase deficiency with epilepsy and cognitive impairment; associated with developmental disorders
PMID: 35850019
2
ABAT is among genes encoding proteins that function in maintaining balanced mitochondrial nucleotide pools essential for mtDNA maintenance
PMID: 28215579
⚠Limited data available β€” This gene has 2 indexed publications. Summary and analysis may be incomplete.
Disease Associationsβ“˜21
Gamma-aminobutyric acid transaminase deficiencyOpen Targets
0.80Strong
GABA aminotransaminase deficiencyOpen Targets
0.73Strong
infantile spasmsOpen Targets
0.56Moderate
mitochondrial DNA depletion syndromeOpen Targets
0.54Moderate
epilepsyOpen Targets
0.51Moderate
Focal impaired awareness seizureOpen Targets
0.47Moderate
complex partial epilepsyOpen Targets
0.46Moderate
developmental and epileptic encephalopathyOpen Targets
0.42Moderate
partial epilepsyOpen Targets
0.37Weak
developmental disabilityOpen Targets
0.37Weak
genetic disorderOpen Targets
0.34Weak
cardiomyopathyOpen Targets
0.27Weak
systemic inflammatory response syndromeOpen Targets
0.27Weak
placental retentionOpen Targets
0.24Weak
hepatitis B virus infectionOpen Targets
0.22Weak
cocaine dependenceOpen Targets
0.22Weak
tuberous sclerosisOpen Targets
0.20Weak
BlindnessOpen Targets
0.19Weak
genetic developmental and epileptic encephalopathyOpen Targets
0.18Weak
sign or symptomOpen Targets
0.18Weak
GABA-transaminase deficiencyUniProt
Pathogenic Variants39
NM_020686.6(ABAT):c.631C>T (p.Leu211Phe)Pathogenic
Gamma-aminobutyric acid transaminase deficiency|Inborn genetic diseases
β˜…β˜…β˜†β˜†2024β†’ Residue 211
NM_020686.6(ABAT):c.22C>T (p.Gln8Ter)Pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜…β˜†β˜†2024β†’ Residue 8
NM_020686.6(ABAT):c.275G>A (p.Arg92Gln)Pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜…β˜†β˜†2024β†’ Residue 92
NM_020686.6(ABAT):c.168+1G>APathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜…β˜†β˜†2023
NM_020686.6(ABAT):c.405del (p.Pro136fs)Pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜†β˜†β˜†2026β†’ Residue 136
NM_020686.6(ABAT):c.1031G>A (p.Trp344Ter)Pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜†β˜†β˜†2025β†’ Residue 344
NM_020686.6(ABAT):c.1145G>A (p.Trp382Ter)Pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜†β˜†β˜†2025β†’ Residue 382
NM_020686.6(ABAT):c.658_659del (p.Arg220fs)Pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜†β˜†β˜†2025β†’ Residue 220
NM_020686.6(ABAT):c.199-2A>CLikely pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜†β˜†β˜†2025
NM_020686.6(ABAT):c.991G>A (p.Gly331Arg)Likely pathogenic
not provided
β˜…β˜†β˜†β˜†2024β†’ Residue 331
NM_020686.6(ABAT):c.571C>T (p.Gln191Ter)Likely pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜†β˜†β˜†2024β†’ Residue 191
NM_020686.6(ABAT):c.604-1G>ALikely pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜†β˜†β˜†2023
NM_020686.6(ABAT):c.540+2T>GLikely pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜†β˜†β˜†2023
NM_020686.6(ABAT):c.1094_1097dup (p.Lys367fs)Likely pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜†β˜†β˜†2023β†’ Residue 367
NM_020686.6(ABAT):c.615C>A (p.Cys205Ter)Likely pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜†β˜†β˜†2023β†’ Residue 205
NM_020686.6(ABAT):c.145G>T (p.Glu49Ter)Likely pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜†β˜†β˜†2023β†’ Residue 49
NM_020686.6(ABAT):c.817-1G>TLikely pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜†β˜†β˜†2023
NM_020686.6(ABAT):c.1297del (p.Arg432_Val433insTer)Likely pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜†β˜†β˜†2023β†’ Residue 432
NM_020686.6(ABAT):c.864dup (p.Gly289fs)Likely pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜†β˜†β˜†2023β†’ Residue 289
NM_020686.6(ABAT):c.181C>T (p.Gln61Ter)Likely pathogenic
Gamma-aminobutyric acid transaminase deficiency
β˜…β˜†β˜†β˜†2023β†’ Residue 61
View on ClinVar β†—
Drug Targets1
VIGABATRINApproved
Gamma-amino-N-butyrate transaminase inhibitor
Related Genes
ALDH5A1Protein interaction99%GADL1Protein interaction95%ALDH3B1Protein interaction95%ALDH3B2Protein interaction95%GATMProtein interaction95%ALDH3A1Protein interaction94%
Tissue Expression6 tissues
Liver
100%
Brain
23%
Heart
7%
Ovary
4%
Bone Marrow
3%
Lung
1%
Gene Interaction Network
Click a node to explore
ABATALDH5A1GADL1ALDH3B1ALDH3B2GATMALDH3A1
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted Β· UniProt P80404
View on AlphaFold β†—
Constraintβ“˜
LOEUFβ“˜
0.87LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.68 [0.53–0.87]
RankingsWhere ABAT stands among ~20K protein-coding genes
  • #7,836of 20,598
    Most Researched58
  • #996of 1,025
    FDA-Approved Drug Targets1
  • #1,570of 5,498
    Most Pathogenic Variants39
  • #7,683of 17,882
    Most Constrained (LOEUF)0.87
Genes detectedABAT
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
Empagliflozin in Patients with Chronic Kidney Disease.
PMID: 36331190
N Engl J Med Β· 2023
1.00
2
Long-Term Effects of Empagliflozin in Patients with Chronic Kidney Disease.
PMID: 39453837
N Engl J Med Β· 2025
0.90
3
Mitochondrial DNA maintenance defects.
PMID: 28215579
Biochim Biophys Acta Mol Basis Dis Β· 2017
0.80
4
Genetic variations in GABA metabolism and epilepsy.
PMID: 35850019
Seizure Β· 2022
0.70
5
Effects of empagliflozin on progression of chronic kidney disease: a prespecified secondary analysis from the empa-kidney trial.
PMID: 38061371
Lancet Diabetes Endocrinol Β· 2024
0.60