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liposomal WT1 siRNA
- Development stage
- Preclinical
- Lead developer
- University of Missouri
- 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, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Inhalation
01
Overview
Liposomal WT1 siRNA is a preclinical-stage RNA interference (RNAi) therapeutic consisting of a small interfering RNA (siRNA) sequence designed to silence the Wilms' Tumor 1 (WT1) gene, encapsulated within a neutral liposomal nanocarrier composed of 1,2-dioleoyl-sn-glycero-3-phosphocholine (DOPC). WT1 is a transcription factor that is frequently overexpressed in various malignancies, including melanoma and certain leukemias, where it acts as an oncogene promoting cell survival, proliferation, and metastasis. This specific formulation is optimized for respiratory administration via inhalation to achieve localized delivery to the lungs, particularly for the treatment of lung metastatic melanoma. In murine models, the therapy has demonstrated significant reduction in WT1 protein expression, leading to decreased tumor weight and improved survival by inducing G1 cell-cycle arrest and apoptosis in cancer cells.
- Other names
- liposomal Wilms' Tumor 1 siRNADOPC liposomal WT1 siRNAliposomal siRNA targeting WT1
02
Targets
WT1 (Wilms tumor protein)
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