A life-saving therapy has been developed at the Telethon Institute in Milan.
The various approaches to gene therapy developed by researchers at SR-Tiget have proved to be effective for treating genetic diseases that affect children. Studies are already underway which will lead to new clinical trials for the treatment of other diseases.
This all-Italian success story is allowing families around the world to 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.
SR-Tiget has demonstrated the effectiveness of gene therapy in the treatment of ADA-SCID, a serious immune system disorder. It has also produced extremely satisfying results regarding the treatment of children with metachromatic leukodystrophy, a neurodegenerative disease, and Wiskott-Aldrich syndrome, a serious congenital form of immunodeficiency. Further trials are in the pipeline in the quest for a treatment for two genetic blood disorders.
SR-Tiget was the first academic laboratory in Europe to be certified as a GLP (Good Laboratory Practice)-compliant test facility, attesting to its conformity with international good laboratory practice standards developed to ensure the quality of organisational processes.
SR-Tiget was established as a centre of excellence to host all stages of basic and clinical research in the field of gene and cell therapy, in order to develop new treatment protocols for genetic diseases.
Strimvelis: gene therapy for ADA-SCID
The therapy developed at SR-Tiget for the treatment of ADA-SCID (a rare primary immunodeficiency) is one of the first examples of effective gene therapy in the world. This result was made possible thanks to our ability to be forward-thinking and to recognise the benefits of gene therapy.