the San Raffaele Telethon Institute for Gene Therapy - SR-TIGET

A therapy that may save many lives was developed at the Telethon institute in Milan.

The various approaches to gene therapy developed by the researchers at SR-Tiget display efficacy against 3 genetic diseases among infants. Experiments are already starting up for the treatment of other diseases. To families worldwide, this 100% Italian work provides the hope that one day they, too, will find the key to the health and wellbeing of their children.


  • The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) was founded in 1995.
  • Tiget hosts 16 groups of researchers working under the supervision of Luigi Naldini. Its scientific staff numbers 140 persons.
  • During the last tax year, we invested more than 9.5 million euros in this institute. This is a part of the approximately 65 million we have invested since the year in which the institute came into being.
  • SR-Tiget has demonstrated that gene therapy is efficacious in the treatment of ADA-SCID, a serious immune system disorder. SR-Tiget is also most satisfied at the results of its work on children presenting metachromatic leukodystrophy, a neurodegenerative disease, and Wiskott-Aldrich syndrome, a serious congenital immunodeficiency disorder. Experimentation shall soon also start up as art of the search for a treatment for two genetic diseases of the blood.
  • SR-Tiget is the first academic laboratory in Europe to be certified as a GLP-compliant test facility − Centro di Saggio Glp (Good Laboratory Practice) − attesting to conformity with the international good laboratory practice standards developed to ensure organisational process quality.

SR-Tiget was founded in order to provide a centre of excellence to host all stages of basic and clinical research in the field of gene and cellular therapy, in order to develop new treatment protocols for genetic diseases.

SR-Tiget, in Milan, is a world leader for gene therapy

The therapy developed at SR-Tiget against ADA-SCID (a rare primitive immunodeficiency condition) is one of the world’s first instances of efficacious gene therapy. This result was made possible thanks to the far-sightedness of our organisation, which immediately expressed its belief in the benefits of gene therapy.