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CNB-nanoplasmid gene therapy is an experimental non-viral gene delivery system designed to target pancreatic beta cells for the treatment of Type 1 Diabetes (T1D). The system utilizes cationic nanobubbles (CNBs) as carriers for therapeutic nanoplasmids (NPs). Delivery is achieved through "Nano2transfection," a technique involving focused ultrasound-mediated sonotransfection, where high-energy sonication induces cavitation of the nanobubbles to enable site-specific gene transfer. The primary goal of this immunomodulatory gene therapy is to reduce inflammation and promote islet recovery and function in the early stages of T1D. Preclinical studies in NOD/LtJ mouse models have demonstrated that intravenous administration of these complexes, combined with targeted ultrasound, successfully achieves localized gene delivery to pancreatic islets without inducing significant tissue damage or inflammation.
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