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Target intelligence / Profile preview
Solute carrier family 25 member 13 (SLC25A13)
- Target
- SLC25A13
- Molecular classification
- Transporter, Mitochondrial carrier protein, Solute carrier family (SLC family)
01
Overview
Solute carrier family 25 member 13 (SLC25A13, also known as citrin or AGC2) is a mitochondrial inner membrane transporter that mediates the exchange of aspartate for glutamate and a proton as part of the malate-aspartate shuttle. It is activated by calcium, contains EF-hand calcium-binding domains, and is essential for hepatic amino acid metabolism, nucleotide synthesis, and the urea cycle. Loss-of-function mutations cause citrin deficiency, leading to disorders such as neonatal intrahepatic cholestasis and adult-onset type II citrullinemia, characterized by hyperammonemia and neurological symptoms due to disrupted nitrogen processing. Therapeutic approaches target the metabolic consequences rather than the transporter directly[1][2][3][4][5].
- Other names
- CitrinAGC2 (Aspartate-glutamate carrier 2)CTLN2
02
Mechanism of action
No known drugs with direct mechanism of action against SLC25A13, as therapies target downstream metabolic consequences[3].
03
Biological functions
Amino acid transport (aspartate/glutamate antiporter)Malate-aspartate shuttleUrea cycle regulationGluconeogenesisNucleotide synthesisCellular energy metabolism
04
Disease associations
Metabolic disease (Citrin deficiency, including type II citrullinemia and neonatal intrahepatic cholestasis caused by citrin deficiency)Liver diseaseNeurological disorders associated with ammonia toxicity
05
Safety considerations
Disruption of SLC25A13 function leads to toxic accumulation of ammonia and citrulline, causing neurological impairment and liver dysfunction in affected individuals[2][3][4].Loss-of-function mutations result in severe metabolic syndromes, especially in neonates and adults with citrin deficiency[2][3][4].
06
Biomarkers
Plasma citrulline (elevated in citrin deficiency)Plasma ammonia (elevated due to urea cycle disruption)Genetic mutation analysis of SLC25A13[2][3][4]
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