Drug intelligence / Profile preview

Dock1 shRNA

Development stage
Preclinical
Lead developer
Versiti Blood Research Institute
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

Dock1 shRNA is a short hairpin RNA-based therapeutic candidate designed to knock down the expression of Dedicator of cytokinesis 1 (DOCK1), a guanine nucleotide exchange factor (GEF) for Rac1 and Rac2. In the context of acute myeloid leukemia (AML) harboring NPM1 and cohesin mutations, DOCK1 is uniquely upregulated and promotes leukemic cell survival and growth through the activation of the Rac2 pathway. Preclinical studies have demonstrated that silencing DOCK1 using shRNA leads to reduced cell growth, impaired adhesion, and increased apoptosis in specific leukemic cell lines, as well as prolonged latency in transplant models. This approach highlights DOCK1 as a precision oncology target for specific genetic subsets of AML.

Other names
DOCK1 shRNADOCK-1 shRNADOCK 1 shRNAshDOCK1shDOCK-1shDOCK 1
02

Targets

DOCK1 (Dedicator of cytokinesis protein 1)

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