Arseniy’s story is the story of a child who has rediscovered that he can take his future into his own hands − thanks to our research. Born in 2009 in Russia, Arseniy soon displayed the symptoms of a rare genetic disease − the Wiskott-Aldrich syndrome, a disease presenting an immune system deficiency. He constantly requires hospitalisation and must stay away from other children. His daily life is a constant struggle against allergies, haemorrhages and even minor infections which, for him, may be dangerous. He can’t play in the open air with his friends or stroke a cat, as children normally do. Two years after his disease had been diagnosed, a German doctor told Arseniy’s parents that an innovative therapy − gene therapy − had been developed at the Istituto San Raffaele Telethon (Tiget) in Milan. In March 2012, after his long journey from Russia to Italy, Arseniy became a part of the experimentation undertaken by the Telethon institute.

Telethon immediately concluded that the Tiget discovery had great potentials, and decided to finance the work of the researchers and their clinical trials regarding Wiskott-Aldrich and metachromatic leukodystrophy. Arseniy is just one of the children for whom gene therapy provides hope. To date, more than 30 patients have been subjected to this highly innovative treatment. The Wiskott-Aldrich syndrome and metachromatic leukodystrophy are just two of the genetic diseases that can now be treated with the Naldini therapy. A small donation from you will provide researchers and many other patients with the answers they’re looking for. Your donation provides hope to many families.


Thanks to the generosity of millions of Italians, an Italian institute has been fully engaged since 1995 in gene therapy research and development. Gene therapy is an innovative technique, with fascinating potentials for the treatment of various diseases. Thanks to our unflagging efforts, Italy has become a leader in this field.

The Wiskott-Aldrich syndrome is a rare genetic disease characterised by immune deficiency. In April 2010, at the Istituto Telethon in Milan, the world’s first trial began involving experimentation with human beings to provide a cure for this disease. The trial is still ongoing. The therapeutic protocol includes removal of stem cells from the patients’ medulla ossium. The stem cells are modified in the laboratory by introduction of the vector containing the therapeutic gene. The stem cells are then re-introduced into the organism.