Drug intelligence / Profile preview

OTUD1 shRNA

Development stage
Preclinical
Lead developer
University of Ostrava
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
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Overview

OTUD1 shRNA is a short hairpin RNA (shRNA) construct designed to silence the expression of the OTU domain-containing protein 1 (OTUD1), a plasma cell-specific deubiquitinase. In the context of multiple myeloma (MM) research, OTUD1 has been identified as a critical posttranslational regulator of immunoglobulin (Ig) assembly and stability. Suppression of OTUD1 via shRNA leads to reduced intracellular Ig levels, increased cell proliferation, and the induction of resistance to proteasome inhibitors such as bortezomib. Mechanistically, OTUD1 stabilizes the endoplasmic reticulum (ER) redox protein PRDX4; its knockdown disrupts the OTUD1-PRDX4 axis, leading to a decreased load of misfolded proteins and reduced sensitivity to ER stress-inducing therapies. This tool is primarily used in preclinical studies to validate OTUD1 as a therapeutic target and biomarker for proteasome inhibitor response.

02

Targets

OTU domain-containing protein 1 (OTUD1) mRNA

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