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spherical nucleic acid targeting microRNA-99b
- Development stage
- Preclinical
- Lead developer
- Northwestern University
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous (most Common In Preclinical/clinical Studies)
01
Overview
Spherical nucleic acid targeting microRNA-99b is a nanostructured therapeutic composed of a nanoparticle core densely functionalized with oligonucleotides specifically designed to bind and inhibit microRNA-99b. Spherical nucleic acids (SNAs) are characterized by their three-dimensional architecture, which imparts unique properties such as enhanced cellular uptake, stability against nuclease degradation, and the ability to cross biological barriers like the blood-brain barrier without transfection agents[3][5][6]. By delivering antisense or RNA interference (RNAi) sequences that target miR-99b, this SNA can modulate gene expression for therapeutic purposes. The mechanism of action involves sequence-specific binding to miR-99b, leading to its inhibition and subsequent regulation of downstream gene targets implicated in disease processes. SNAs have been developed primarily at Northwestern University and are being explored for various indications including cancer and genetic diseases[3][4][6].
- Other names
- SNA targeting microRNA-99bSpherical nucleic acid anti-miR-99b
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