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Exo-U2-Dox
- Development stage
- Preclinical
- Lead developer
- Southern Medical University
- Modality
- RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules, DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
**Exo-U2-Dox** is a preclinical, glioblastoma-targeted doxorubicin delivery construct composed of mesenchymal stem cell-derived exosomes surface-functionalized with the GBM-targeting U2 aptamer and loaded with doxorubicin. The engineered exosomes are designed to cross the blood-brain barrier and preferentially accumulate in EGFRvIII-positive glioblastoma. U2-mediated inhibition of EGFRvIII autophosphorylation promotes NLRP3 inflammasome-associated pyroptosis, while delivered doxorubicin contributes DNA damage; in preclinical models, the construct enhanced X-ray radiosensitivity through reduced 53BP1 expression and attenuation of ATM/Chk2 DNA-damage signaling. It has been reported in mouse and cell-line glioblastoma models and is not a marketed medicine.
- Other names
- Exo-U2-DOXExo-U-2-DOXExo-U 2-DOX
02
Targets
TOP2A (DNA topoisomerase II)DNA
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