World leaders in
gene therapy

The first achievement by the TIGET - Telethon Institute for Gene Therapy in Milan was the successful completion of a clinical trial for Ada-Scid, a severe congenital immunodeficiency. Results of the study, published in the New England Journal of Medicine in 2009, represented a major milestone in the history of gene therapy.

In 2013 the institute’s contribution to establishing gene therapy as a promising innovative therapeutic approach for genetic diseases was definitively acknowledged by the international scientific community with the success of the clinical trials on another immunodeficiency, Wiskott-Aldrich syndrome (Was), and a metabolic disease leading to severe neurodegeneration in children, metachromatic leukodystrophy (Mld).

The results from the trials, published in two independent back-to-back articles in Science, are universally renowned as a breakthrough achievement with significant potential impact beyond the treatment of rare pediatric diseases previously without a cure, like Was and Mld.

The novely of the approach, developed by Luigi Naldini, director of the Telethon Institute for Gene Therapy, lies in the extremely efficient and safe viral vectors, called “lentiviral”, engineered from the the Hiv virus. In fact, preclinical research carried out at the institute is showing promising results towards a future application of lentiviral vectors to the treatment of common diseases such as cancer.