Target intelligence / Profile preview

Ubiquitin specific peptidase 1 (USP1)

Target
USP1
Molecular classification
Enzyme, UBP family, Peptidase C19, Deubiquitinating enzyme (DUB)
01

Overview

USP1 is a deubiquitinating enzyme (DUB) belonging to the UBP family. It plays a crucial role in DNA damage response and repair by regulating the ubiquitylation status of key proteins like FANCD2, FANCI, and PCNA. USP1 is involved in the Fanconi anemia pathway and autophagy regulation. It is a therapeutic target in cancer, particularly in BRCA-mutant tumors, where its inhibition shows synthetic lethality with PARP inhibitors.

02

Mechanism of action

Inhibition of USP1 activity leads to accumulation of ubiquitinated substrates and disruption of DNA repair pathways.

03

Biological functions

DNA damage response/repairProtein catabolismAutophagy regulationRegulation of DNA repairDeubiquitination
04

Disease associations

Cancer progression/resistanceFanconi anemia
05

Safety considerations

Potential for off-target effects due to the broad role of deubiquitinating enzymes.Development of resistance mechanisms.
06

Interacting drugs

Olaparib

1 more in the full profile.

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