Stem cell generation and manipulation by nanoparticle mediated gene transfer for the safe clinical application of gene-modified cells
(FKZ 13N11539 bis 13N11541)


  • apceth GmbH & Co. KG, München
  • Fraunhofer-Institut für Biomedizinische Technik (IBMT), St. Ingbert,
  • Westfälische Wilhelms-Universität Münster (WWU)
  • Clinica Universidad de Navarra (CUN), Pamplona, Spanien
  • Instituto de Biologia Experimental e Tecnológica (IBET), Oeiras, Portugal


Nowadays cells for use in cell and gene therapy are modified by integrating viral vectors, which introduce therapeutic transgenes into the host genome and therefore carry potential safety risks for patients. A promising alternative to viral vectors are synthetic nanoparticle-based systems for gene delivery. The NanoGene project involves five European partners from industry, academia and clinics. The project goal is the development and evaluation of innovative gene-transfer systems based on nanoparticles (NPs) for genetic modification of cells and their safe clinical application.

For this purpose DNA-vector tools for stable cell transfection, such as the PhiC31-integrase and the AAV-Rep system, as well as vectors containing transcription-factors for reprogramming primary somatic cells to iPS have been successfully developed. The packaging of these vectors into biodegradable human serum albumin nanoparticles (HSA-NPs) as well as the uptake of the generated vector-NP complexes into the cells are currently being optimized in vitro. At the same time a strategy for surface-structure modification of HSA-NPs by specific ligands in order to further enhance and specify the uptake into the target cells is being established. The cells used for gene modification by the novel NP-systems are i) mesenchymal stem cells (MSC) for a future gene therapy of cancer , and ii) primary somatic cells for generation of induced pluripotent stem cells.

MSC and iPS produced by gene modification with the NanoGene nanoparticular system will be extensively tested in vitro and in vivo. In addition, in order to obtain a sufficient quantity of MSC for clinical application a robust 3D bioreactor system with optimized culture conditions is being intensively developed. The most suitable microcarriers for hMSC attachment and growth have already been identified.

Weitere Informationen
Dr. Christine Günther
apceth GmbH & Co. KG
Max-Lebsche-Platz 30
81377 München

nach oben Aktualisiert am: 13 Juni 2013.