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SPINAZZOLA THE CHALLENGE OF THE SMALL ORLANDO, suffering from a rare disease, TESTIMONIAL TO WIN ONE OF THE TELETHON

of Cosimo Forina

Today, Friday 17, the program "Life Direct Rai 1, from 16, as guests of the small Telethon praying Barrasso Spinazzola accompanied by his family. Orlando suffered from "Leber congenital amaurosis," a serious disease that affects the retina early in life and leads progressive loss of vision, has already been testimonial last year's Telethon, embraced at the start of the telethon also by the President of the Chamber on.
Gianfranco Fini . He is one of the twelve patients in the world, the smallest, which was applied to the largest dose, five Italians, on which the gene therapy experiments improve vision.
EARLY INTERVENTION
"The first action - tell Dad Joseph
, psychiatrist, and mother- Adriana Telethon made through research has been carried out in the right eye in June 2009. In the future it is expected that the the left eye next year also will suffer the same type of assistance thus completing this phase of the experimental study of the development of gene therapy. Participation in the transmission is designed to raise public awareness to support research, provide hope, communicate that with the help of all genetic diseases are tackled and defeated. " The trial involves the International Amaurosis Leber's Children's Hospital of Philadelphia where Orlando has undergone the first surgery Telethon Institute of Genetics and Medicine (Tigem) of Naples and, also in capital of Campania, the Department of Ophthalmology at the Second University of studies (Sun). In Naples, Orlando, has found its first angels of light in the researchers Tigem: Alberto Auricchio, Enrico Maria Surace
and Sandro Banfi
, which two years are evaluating the safety and efficacy of gene therapy. molecular strategy In the same city was put in place a real "molecular approach" to correct the defect in the genes that cause eye diseases such as RPE65, one of the leaders of dell'amaurosi Leber. The clinical team is led by the Department of Ophthalmology Francesca Simonelli, Francesco Testa part and Septimus Smith dealing to identify patients on which to experiment. Last year was the 2 June 2009, to tell via e-mail to the Gazette almost live from the Children's Hospital of Philadelphia, among the five best hospitals in the United States, the record of intervention Father Joseph, "Orlando was made. The operation was coordinated by Dr. Kathy Marshall
that followed step by step throughout the development of controls, accompanied by researcher Dr. Jean Bennett
, wife of dr. Maguire who has literally performed the operation that consisted of an injection in the right eye of an attenuated virus as vectors (A kind of taxi) that correct the genetic code should replace the corrupted. Healthy copies of the gene were injected into the diseased retina, where photoreceptors enter the cells, which transform visual stimuli into nerve impulses, restoring the function of the eye. " After returning from the United States, Orlando, like other patients who underwent surgery, was subjected to a close and continuous monitoring. POSITIVE RESULTS And here is the first positive news, it is understood by the journal The Lancet, where the trial was published: "How, after examination on a regular basis following the intervention, none of the patients reported so far toxic effects significant, confirming how the procedure is safe. At the same time, visual function tests clearly indicate that in all there was a partial recovery of vision, especially in younger patients. " "This - Francesca Simon says - is the most important result: the more the gene therapy is initiated early, the higher the probability that the retina of patients is not completely compromised and react positively to treatment." Thanks to the experimentation of Orlando were created new hopes, always by 'The Lancet': an important indication for the future, as pointed out by Alberto Auricchio, "we're going to try to test the effectiveness gene therapy not only for other forms of amaurosis, but also for other genetic eye diseases such as Stargardt's disease, for which the Tigem has already obtained orphan drug designation by the Food and Drug Administration and the European Medicines Agency. The eye is certainly an ideal organ for this type of therapy, because it is small, limited and "immunoprivilegiato", which makes possible the administration of low doses of medication and reduces the risk of rejection by the immune system. " Now we have to enjoy the smile of Orlando.