Monica Ricci

Nanowire based intracellular delivery: a versatile tool for gene therapy
Started on October, 2015
Prof. Hiroshi Uji-i (promotor)

email Monica Ricci

In 2006 researchers found a new way to ‘reprogram’ adult specialized cells into stem cells. These so-called induced pluripotent stem cells (iPSCs) were a breakthrough for genetic studies in human cells. The ability to genetically modify human iPSCs holds great promise for gene therapy and other clinical applications. However, while the immortalized human tumour cells lines commonly use at the laboratory level can be edited with almost complete efficiency, much lower success rates are achieved in iPSCs. Using the recent advances in nanotechnology, we propose to develop a new method for the delivery of genetic material in the cellular environment with high delivery efficiency and applicable to all cell types, including stem cells. It has been already shown that nanoscale structures, such as nanotubes and nanowires (NWs), have the ability to penetrate cell membranes with minimal cellular damage. Such nanotechnology provides a powerful new modality for delivering genetic material and other biomolecules and has opened up new opportunities to manipulate living cells. Using NW-based gene delivery the amount of DNA, critical for genetic modification, is easily controlled. The application of NW based gene delivery to reprogram adult cells into iPSCs, modify its genome and induce differentiation into a specific cell type has the potential to drive autologous gene therapy a step closer to clinical applications.