Drug intelligence / Profile preview

shG3BP1

Development stage
Preclinical
Lead developer
Princess Margaret Cancer Centre
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
01

Overview

shG3BP1 is a short hairpin RNA (shRNA) designed to knock down the expression of Ras GTPase-activating protein-binding protein 1 (G3BP1). G3BP1 is a critical protein involved in the nucleation and assembly of stress granules (SGs), which are biomolecular condensates that protect cells under various stress conditions. In the context of oncology, specifically Acute Myeloid Leukemia (AML), G3BP1-mediated stress granules are enriched in leukemic stem cells (LSCs) and contribute to their maintenance, stress adaptation, and chemoresistance. By silencing G3BP1, shG3BP1 impairs stress granule formation, leading to increased apoptosis, enhanced differentiation, and reduced self-renewal capacity of LSCs while sparing normal hematopoietic stem cells. This research-stage tool highlights G3BP1 and stress granule dynamics as potential therapeutic targets in AML.

Other names
G3BP1 shRNAG-3BP1 shRNAG 3BP1 shRNAshort hairpin RNA targeting G3BP1
02

Targets

G3BP1 (Ras GTPase-activating protein-binding protein 1)

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