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Small ubiquitin-like modifier activating enzyme (SAE (also known as SAE1/SAE2 or Aos1/Uba2 for the heterodimeric human complex))

Target: SAE (also known as *SAE1/SAE2* or *Aos1/Uba2* for the heterodimeric human complex)
Molecular classification: Enzyme, Ubiquitin-like modifier activating enzyme, E1 activating enzyme, Post-translational modification enzyme

Overview

The **small ubiquitin-like modifier activating enzyme** is an essential heterodimeric enzyme (SAE1/SAE2 in humans) responsible for initiating the SUMOylation process, a post-translational modification pathway. This enzyme catalyzes the ATP-dependent activation of SUMO proteins (small ubiquitin-like modifiers), forming a high-energy thioester intermediate with the catalytic cysteine before transferring SUMO to the E2 conjugating enzyme (Ubc9). SUMOylation alters the function, localization, and interactions of a wide range of cellular proteins, controlling vital processes including DNA repair, cell cycle progression, transcription, chromatin organization, and cellular stress response. Dysregulation of SUMOylation, and hence of this activating enzyme, is implicated in cancer and other diseases, making it a potential therapeutic target[1][2][5][4].

Other names: SUMO-activating enzymeSAE1/SAE2 (human heterodimer)Aos1/Uba2 (yeast)SUMO E1SUMO-activating enzyme E1Ubl activating enzyme for SUMO

Mechanism of action

Inhibition of SUMOylation by blocking SUMO activation (adenylation and thioester formation) Prevention of SUMO transfer to E2 and downstream conjugation to target proteins

Biological functions

Protein post-translational modification (SUMOylation)Regulation of DNA damage repairRegulation of gene expression/transcriptionControl of chromatin structureCell cycle control (mitosis, replication)Stress response regulation

Disease associations

CancerNeurodegenerative disease (linked via UBA1 pathway, functional analogy)Disorders of genome maintenance and DNA repairOther (cell cycle and proliferation abnormalities, stress response syndromes)

Safety considerations

Inhibition may affect essential processes including DNA repair and cell cycle progression, potentially resulting in cytotoxicity in normal proliferating tissuesBroad suppression may lead to immune or stress response deficiencies

Interacting drugs

Direct, clinically approved inhibitors are in preclinical/early research; specific chemical probes and small molecules are being used experimentally, but no widely recognized drugs approved.

1 more in the full profile.

Biomarkers

SUMOylation levels (as functional output indicator)SAE1 or SAE2 expression (proxy for enzyme activity in disease states)

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