The San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), stems from a collaboration initiated in 1995 between Telethon and the Fondazione San Raffaele del Monte Tabor. The laboratories are located within the DiBiT, San Raffaele Hospital in Milan.
HSR-TIGET focuses on basic research and on experimental protocols for gene therapy of inherited diseases, in particular, lysosomal diseases and congenital immunodeficiencies.
In the past few years a series of successful milestones were reached both in basic and gene therapy research projects.
One of HSR-TIGET’s most notable results is represented by the Clinical Trial for the immunodeficiency ADA-SCID. Ten paediatric patients affected by hereditary immunodeficiency were cured with a gene therapy protocol that has enabled them to produce the missing enzyme and to reconstitute their immune system.
HSR-TIGET’s scientific staff counts 74 people, led by Maria Grazia Roncarolo together with Luigi Naldini.
HSR-TIGET’s funding is renewed every five years through a non competitive call and a rigorous revision process. Each HSR-TIGET scientist presents her/his projects and is evaluated according to peer review criteria. A select group of Scientific Committee members also performs a site visit of the Institute. The final decision is taken during the plenary meeting, where the projects are discussed singularly and only the best ones are selected for funding.
Since 1995 Telethon has invested approximately 27,000,000 Euros in HSR-TIGET.The last site visit took place in November 2005, and funding of approximately 3,000,000 Euros was granted annually for the next five years (2005-2010).
As an additional proof of its scientific worth, HSR-TIGET can boast several grants funded by other major financing agencies such as the European Community, the NIH and other Italian research Institutions.
For more information: TIGET