Target intelligence / Profile preview

Cationic amino acid transporter 3 (CAT-3)

Target
CAT-3
Molecular classification
Transporter, Solute carrier family 7 (SLC7) member, Membrane protein
01

Overview

Cationic amino acid transporter 3 (CAT-3) is a sodium-independent membrane transporter responsible for the uptake of cationic amino acids such as arginine, lysine, and ornithine, and is encoded by the SLC7A3 gene in humans. It is primarily expressed in the brain and plays a crucial role in neuronal physiology, modulating signalling pathways including nitric oxide synthesis (by controlling arginine availability) and the mammalian target of rapamycin (mTOR) pathway, which is vital for neuronal growth, differentiation, and synaptic plasticity. Variants in CAT-3 have been implicated in neurodevelopmental disorders, such as autism spectrum disorder, as well as being of interest in other diseases due to the transporter's fundamental role in cell metabolism. While no approved drugs directly target CAT-3, it is of therapeutic interest, with the broader family of amino acid transporters being explored as drug targets, notably in cancer and neurological conditions. The main therapeutic challenge is the essential role of cationic amino acid transport in various cell types, making safety and selectivity a concern for any potential targeted interventions.

Other names
SLC7A3ATRC3CAT3Cationic amino acid transporter y+Solute carrier family 7 member 3
02

Mechanism of action

Drugs acting on CAT-3 would generally function as competitive inhibitors of cationic amino acid transport, reducing intracellular supply of substrates like arginine, and thereby affecting downstream processes such as NO synthesis and mTOR signalling

03

Biological functions

Uptake and transport of cationic amino acids (arginine, lysine, ornithine)Regulation of nitric oxide (NO) synthesis via arginine availabilityModulation of mammalian target of rapamycin (mTOR) pathway, impacting neuronal development and plasticityResponds to NMDA receptor activation in neurons
04

Disease associations

Neurological disorders (impairment affects neuronal signalling, plasticity, and development)Potential modifier in autism spectrum disorder (via mTOR pathway dysfunction)General: Disruption may play a role in disorders involving arginine/NO/mTOR pathwaysEmerging interest in cancer and other diseases due to broader role of amino acid transporters
05

Safety considerations

Broad inhibition of cationic amino acid transport may disrupt essential cellular functions, particularly in neurons (via impact on NO, mTOR, and neurotransmitter regulation)Risks include neurodevelopmental effects, impaired cell signalling, and metabolic disruptions due to altered amino acid availability
06

Interacting drugs

Arginine (substrate)

3 more in the full profile.

07

Biomarkers

Potential: Expression levels of SLC7A3/CAT-3 in specific tissues (e.g., brain) may serve as biomarkers for neuronal function or disease states involving mTOR pathway dysregulationNo established, clinically validated biomarkers specific to CAT-3 identified

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