FOR 917: Nanoparticle-based Delivery of Innovative Therapies (NanoTarget)

基于基因和细胞的疗法在治疗人类疾病方面具有巨大的潜力。然而，将遗传物质(核酸、病毒和非病毒载体)转移到特定身体区域的特定细胞仍然是成功的基于基因的治疗的主要障碍。此外，许多基于细胞的治疗(例如干细胞)需要在患者的器官中有针对性地定位细胞，以实现治疗细胞在受损器官中的有效积聚。这个研究单位的目标是开发基于纳米颗粒的策略，用于定向转导和定位细胞。我们将重点关注心血管系统，因为它与医学上有很高的相关性。此外，心血管连续体对于通过循环提供治疗--无论是遗传物质和/或细胞--来说是最重要的。为了实现对遗传物质和转导细胞的非侵入性靶向，将使用具有超顺磁性的纳米颗粒(磁性纳米颗粒，MNPs)。使用MNPs的最大优点是这些颗粒和它们的货物以及含有MNP的细胞可以通过磁场引导。

Gene- and cell-based therapies have great potential for the treatment of human diseases. However, targeting the transfer of genetic material (nucleic acids, viral and non-viral vectors) to specific cells in certain, defined body areas is still a major hurdle for successful gene-based therapies. In addition, targeted positioning of cells in organs of the patient would be required for many cell-based therapies (e.g. stem cells) to achieve efficient accumulation of therapeutic cells in damaged organs. The aim of this Research Unit is the development of nanoparticle-based strategies for targeted transduction and positioning of cells. We will focus on the cardiovascular system, because of its high medical relevance. Furthermore, the cardiovascular continuum is of outmost importance for the delivery of therapies - be it genetic material and/or cells - via the circulation. To achieve non-invasive targeting of genetic material and transduced cells, nanoparticles will be used that are superparamagnetic (magnetic nanoparticles, MNPs). The use of MNPs has the big advantage that these particles and their cargo as well as MNP containing cells can be guided by magnetic fields.