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Non-invasive nanotransducer for in vivo gene therapy

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Nanotechnology is expected to have a major impact on biomedical research, leading to new types of diagnostic and therapeutic tools. One focus in nanobiotechnology is the development of non-viral 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.

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 functionalised with a ligand, able to bind a receptor expressed by the target cells to be transfected. 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) cells up -take the vectors via endocytosis and 2) genes are transferred in the cell membrane via electroporation (i.e. via permeabilization of cell membrane by application of short-duration electric field pulses).

In the second case, current impulses could be generated in CNTs via electromagnetic fields, by exploiting t he properties of CNTs like antennas. In order to prove these paradigms, in vivo validation of the nanotransducer vectors will be performed. A specific neurological disorder will be treated to demonstrate their therapeutic potential. The implementation of t his proposed solution requires a multidisciplinary approach and the integration of expertise in the fields of nanotechnologies, biotechnologies, communication technologies and neuroscience.

Aufforderung zur Vorschlagseinreichung

FP6-2004-NMP-TI-4
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SCUOLA SUPERIORE DI STUDI UNIVERSITARI E DI PERFEZIONAMENTO SANT'ANNA
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PIAZZA MARTIRI DELLA LIBERTA' 33'
PISA
Italien

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