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small interfering RNA targeting PAK1
- Development stage
- Preclinical
- Lead developer
- Dharmacon
- 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, Intratumoral
01
Overview
Small interfering RNA targeting PAK1 (siPAK1) is an experimental therapeutic agent designed to silence the expression of p21-activated kinase 1 (PAK1) via the RNA interference (RNAi) pathway. PAK1 is a member of the Group I Paks, acting as a key effector for the small Rho GTPases Rac1 and Cdc42, and is frequently overexpressed in various cancers, including endometrial, ovarian, and breast cancers. By degrading PAK1 mRNA, this siRNA reduces protein levels, leading to the inhibition of cell cycle progression (through down-regulation of cyclin D1) and modulation of steroid receptors like ERα and PR. In preclinical models of endometrial cancer, siPAK1 has demonstrated the ability to significantly reduce cell proliferation, particularly in type I endometrioid carcinomas.
- Other names
- PAK1 siRNAPAK-1 siRNAPAK 1 siRNAsiRNA-PAK1siRNA-PAK-1siRNA-PAK 1
02
Targets
PAK1 (p21-activated kinase 1)
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