Drug intelligence / Profile preview

WAVE1-siRNA

Development stage
Preclinical
Lead developer
National Institute of Neurological Disorders and Stroke
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
01

Overview

WAVE1-siRNA is a small interfering RNA (siRNA) designed to silence the expression of WASP-family verprolin homologous protein 1 (WAVE1). WAVE1 is a downstream effector of small GTPases that regulates the actin cytoskeleton and modulates cell motility and morphology. In glioblastoma (GBM), WAVE1 is often upregulated compared to normal brain tissue. By targeting WAVE1 mRNA for degradation, this siRNA inhibits the proliferation, migration, and self-renewal of patient-derived primary GBM neurospheres. Research indicates that WAVE1 knockdown exerts a cytostatic effect rather than an apoptotic one, suggesting its potential as a therapeutic strategy to limit tumor progression and invasiveness in high-grade gliomas.

Other names
WASP-family verprolin homologous protein 1 siRNAWAVE1 knockdown siRNAWAVE-1 knockdown siRNAWAVE 1 knockdown siRNA
02

Targets

Wiskott-Aldrich syndrome protein family member 1 (WASF1) mRNA

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