Preclinical Nanomedicine

The working group of Preclinical Nanomedicine is focused on the development and evaluation of new therapeutic approaches based on nanoparticles. Nanoparticles are a flexible transport platform for various applications such as tumour targeting, gene therapy or neurodegenerative diseases. Biocompatible and biodegradable proteins and polymers such as human serum albumin (HSA) or the polymer polylactic acid (PLA) form the backbone of the developed nanoparticles. For the directed targeting, the nanoparticles are modified with cell-specific ligands. The specific nanoparticles transport drugs directly to the target cells without impairing or harming neighbouring cells. Thus, side effects of e. g. toxic drugs such as cytostatic agents for the tumour targeting can be reduced.
For the genetic modification of cells, the biocompatible nanoparticles are loaded with DNA, RNA or small molecules. The aim is to develop efficient gene delivery systems for primary and stem cells to replace viral-based delivery systems in gene and cell therapy.

The directed targeting is not only used for tumour targeting or genetic modification of cells but also for challenging the crossing of biological barriers, which the nanoparticles have to pass depending on the route of administration. Various barrier models such the blood-brain barrier (bbb), intestinal barrier and skin barrier have been established for the analysis of nanoparticular formulations. These barrier models are complex cell culture models like the blood-brain barrier consisting of primary brain capillary endothelial cells in a Transwell® system which is used for the identification of new drugs for neurodegenerative diseases.

The continual development and optimization of the cell culture models are a central part of our research. Therefore, the barrier models are set-up with specific cell types differentiated from human pluripotent stem cells (hiPSCs). Further, hiPSCs derived of somatic cells from patients are used to develop disease-specific cell models or organoids for drug screening or disease studies.