Video Link: https://www.youtube.com/watch?v=STarrtjF8XE

16-01-2012 03:57 http://www.telethon.it - Gene therapy is the technique that allows the prevention and treatment of diseases thanks to DNA transfer. In the case of genetic diseases in which one gene is defective or absent, gene therapy basically consists in transferring a functioning version of the gene into a patient's organism so as to remedy the defect. The idea at the basis of gene therapy is a simple one, but implementing it means negotiating an obstacle course. The more delicate aspects include the safety of the procedure, the efficiency of the transfer and the immune system's reaction, which can result in the elimination of the genetically modified cells and annul the effects of the treatment. The first success in gene therapy was achieved in Italy in 2002, thanks to Telethon research, curing the first two children suffering from ADA-SCID and therefore born with no immune defences. Today there are 14 children who have been totally cured and Telethon is working on making gene therapy available for everyone. To this one must add the success achieved by researchers at the Telethon Institute of Genetics and Medicine (TIGEM) at Naples' Second University and at the Children's Hospital in Philadelphia, with the treatment of 12 patients suffering from Leber's congenital amaurosis, the disease of the retina that leads to blindness.

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Gene therapy, a totally Italian success - Video