Target intelligence / Profile preview

Adenylosuccinate synthetase isozyme 1 (ADSS1)

Target
ADSS1
Molecular classification
Enzyme (EC 6.3.4.4), Ligase (specifically forms carbon-nitrogen bonds)
01

Overview

Adenylosuccinate synthetase isozyme 1 is an enzyme that catalyzes the GTP-dependent conversion of inosine monophosphate (IMP) and aspartic acid into adenylosuccinate during de novo AMP biosynthesis. It plays a critical role in regulating cellular levels of adenine nucleotides through participation in the purine nucleotide cycle—a pathway important for energy balance especially in muscle tissue. The human gene encoding this protein is ADSS1; mutations can result in rare inherited myopathies due to impaired purine metabolism. Structurally, it functions as a homodimer with each subunit containing characteristic beta-sheets and alpha-helices typical for enzymes binding nucleotides such as GTP[3][5][6].

Other names
Adenylosuccinate synthetase-like 1Adenylosuccinate synthetase, basic isozymeAdenylosuccinate synthetase, muscle isozymeAdSS 1ADSSL1AMPSase 1IMP--aspartate ligase 1M-type adenylosuccinate synthetase
02

Mechanism of action

Not applicable for approved drugs, but theoretically, inhibition would block AMP synthesis from IMP by preventing GTP-dependent ligation of aspartate to inosine monophosphate[3][10].

03

Biological functions

Purine biosynthesis (catalyzes the first committed step in AMP synthesis from IMP)Regulation of nucleotide levels via the purine nucleotide cycle
04

Disease associations

Other (Defects may be associated with rare inherited myopathies or metabolic disorders; not directly linked to major disease classes like cancer or cardiovascular disease based on current knowledge)
05

Safety considerations

Potential safety concerns would include disruption of purine metabolism leading to impaired energy homeostasis and possible muscle dysfunction, given its role in the purine nucleotide cycle—especially since this isoform is muscle-specific[7].

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