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siRNA-DNApk
- Development stage
- Preclinical
- Lead developer
- National Cancer Institute
- 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, Nanoparticles → Drug Delivery Systems
- Administration
- Intratumoral
01
Overview
siRNA-DNApk is an experimental RNA interference (RNAi) therapeutic designed to enhance the efficacy of radiotherapy in the treatment of glioblastoma (GBM). It consists of a small interfering RNA (siRNA) sequence targeting the DNA-dependent protein kinase (DNApk), encapsulated within (1-aminoethyl)iminobis[N-(oleicylcysteinyl-1-amino-ethyl)-propionamide (ECO) nanoparticles for delivery. Developed through a collaboration between the National Cancer Institute and Case Western Reserve University, the drug works by silencing DNApk, a critical component of the non-homologous end joining (NHEJ) pathway responsible for repairing DNA double-strand breaks. By impairing this repair mechanism, siRNA-DNApk sensitizes glioma cells and glioma stem cells to ionizing radiation, leading to increased cell death. Preclinical studies have demonstrated that intratumoral administration of ECO/siRNA-DNApk complexes effectively reduces target protein levels and enhances radiation response in orthotopic glioblastoma mouse models without significantly affecting normal astrocytes.
- Other names
- siRNA targeting DNApksiRNA targeting DNA-dependent protein kinase
02
Targets
DNA-PK (DNA-dependent protein kinase)
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