Drug intelligence / Profile preview

USP1 siRNA

Development stage
Preclinical
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

USP1 siRNA is a research-grade small interfering RNA (siRNA) designed to silence the expression of Ubiquitin-Specific Protease 1 (USP1), a deubiquitinase enzyme that plays a critical role in DNA damage repair and cell cycle regulation. By targeting and promoting the degradation of USP1 mRNA, this reagent prevents the synthesis of the USP1 protein, thereby inhibiting its ability to deubiquitinate key substrates such as PCNA and FANCD2. This disruption impairs the Fanconi anemia (FA) pathway and translesion synthesis, leading to increased DNA damage and apoptosis in cancer cells. Preclinical studies have utilized USP1 siRNA to explore its therapeutic potential in various malignancies, including glioblastoma, multiple myeloma, and renal cell carcinoma, often demonstrating its ability to sensitize tumor cells to chemotherapy and radiation.

Other names
USP1 small interfering RNAUSP-1 small interfering RNAUSP 1 small interfering RNAsiRNA targeting USP1siUSP1siUSP-1siUSP 1
02

Targets

USP1 (Ubiquitin-specific protease 1)

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