NINIVE
FP6-STRP 033378
Non Invasive Nanotransducer for In Vivo gene thErapy
One focus in nanobiotechnology is the development of nonviral vectors for safe and efficient gene delivery. Progress in the Human Genome Project increases the attractiveness of gene therapy as a therapeutic modality for a host of diseases. Today the methodologies for in vivo gene transfection are still in their infancy. The most promising method is based on the use of viral vectors, but its safety is actually under discussion.Objectives
This project aims at developing a nonviral vector for gene delivery, able of a) gene transfection in vivo and on a large amount of cells, b) local and non invasive therapy, c) frequent and easy medication. This nano-device will be based on a carbon nanotube (CNT). It will be coated with genes to be transferred, and will be functionalized with a ligand, able to bind a receptor expressed by the target cells to be transfected.
NINIVE vector: working method
A solution containing a myriad of vectors will be dispensed locally in the target tissue, e.g. via injection; each of them will localise a target cell and bind it. Two strategies of cell transfection will be investigated: 1) via endocytosis and 2) via electroporation.
Case Studies
In order to prove these paradigms, in vivo validation of the nanotransducer vectors will be performed. Two specific neurological disorder will be treated to demonstrate their therapeutic potential, that are:
Ischemia
Rett Syndrome
Useful links:
EC services
European Society Gene and Cell Therapy
EMEA
Mantained
by: g.ciofani@sssup.it