Target intelligence / Profile preview

Argininosuccinate lyase (ASL)

Target
ASL
Molecular classification
Enzyme, Lyase (specifically a lyase class I family member)
01

Overview

Argininosuccinate lyase is a cytosolic enzyme that catalyzes the reversible cleavage of argininosuccinate to produce arginine and fumarate, a critical step in the urea cycle and arginine biosynthesis[1][3][8][9]. The enzyme functions as a homotetramer, with each monomer contributing to the formation of active sites necessary for catalysis[1][2]. Deficiency or mutations in the *ASL* gene disrupt the urea cycle, leading to toxic ammonia buildup, neurological damage, and the genetic disorder argininosuccinic aciduria[3][5][7]. Argininosuccinate lyase also influences nitric oxide synthesis by supporting arginine availability, making it relevant to vascular biology[3]. Management of ASL deficiency primarily involves dietary control, ammonia-scavenging drugs, and L-arginine supplementation[3].

Other names
ASLArgininosuccinaseArginosuccinaseARLY_HUMANArgininosuccinate lyase (EC 4.3.2.1)
02

Mechanism of action

L-arginine supplementation provides the downstream product that is deficient in ASL deficiency, bypassing the metabolic block caused by reduced enzyme activity.

03

Biological functions

Urea cycle (detoxification of ammonia through production of urea)Arginine biosynthesis (conversion of argininosuccinate to arginine and fumarate)Nitric oxide biosynthesis (through production and transport of arginine)Regulation of blood flow and blood pressure (via arginine-derived nitric oxide)
04

Disease associations

Inborn errors of metabolism (Argininosuccinic aciduria)Hyperammonemia (consequence of ASL deficiency in the urea cycle)Neurological and developmental disorders (secondary to ammonia accumulation)Other (rare hepatic and systemic disorders due to urea cycle defects)
05

Safety considerations

Substrate toxicity (ammonia accumulation is acutely neurotoxic)Need for lifelong metabolic management in genetic disorders (dietary restrictions, risk of metabolic decompensation)Therapeutic challenges in correcting enzyme deficiency and preventing neurological damage
06

Interacting drugs

L-arginine (therapeutic supplementation in deficiency states or argininosuccinic aciduria)
07

Biomarkers

Argininosuccinic acid level (elevated in plasma/urine in ASL deficiency)Hyperammonemia (elevated blood ammonia levels in ASL deficiency)Decreased arginine levels (downstream metabolic effect)

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