Benutzerspezifische Werkzeuge

Selective nanoparticles for application in immunotherapy and gene therapy of malignant diseases (SNaPs)

European Social Fund (ESF) via Sächsische Aufbaubank (SAB)

Cancer medicine has made great progress in recent years. Classical treatment methods, such as surgery, chemotherapy and radiation, often achieve a resection of the primary tumor. It is not uncommon for a tumor to recur after a latency period due to metastatic spread of micrometastases. A modern treatment of these micrometastases or recurrences is the use of human immune cells (immunotherapy) and genes (gene therapy) in the form of "Advanced Therapeutic Medicinal Products" (ATMPs), thus enabling personalized cancer therapy. The biggest hurdle in the production and broad application of ATMPs are technical and regulatory limitations, which make the use of viral and non-viral gene transfer systems costly and particularly time-consuming.

The research project "Selective nanoparticles for application in immunotherapy and gene therapy of malignant diseases (SNaPs)" aims at the development of a selective gene transfer system which allows a simplified, efficient and cost-effective transfer of therapeutic DNA or RNA molecules. The modular structure of the SNaPs is based on the combination of molecular targeting structures (e.g. single chain antibodies, protein ligands) and maltose-modified polypropyleneimine dendrimers, which carry the therapeutic minicircle DNA in the form of a sleeping beauty transposon system. The targeting structure allows selective delivery and effect in the target cell, while reducing off-target effects. The use of minicircle DNA reduces the effective dose and prevents an unwanted toll-like receptor-mediated immune response due to the lack of prokaryotic vector components. The sleeping beauty transposon system enables virus-free integration of therapeutic DNA into the genome of the target cell. This research project has two concrete goals: In the field of immunotherapy, the selective gene transfer of chimeric antigen receptors (CAR) using CD7-specific SNaPs to generate tumor-specific, CAR-modified T lymphocytes and in the field of gene therapy, the targeted transfer of therapeutic genes or DNA molecules into tumor cells.

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