The San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET) was created in 1995 as a joint-venture between the San Raffaele Scientific Institute and the Telethon
Foundation for the implementation of basic and clinical research for genetic diseases.
The mission of the Institute is to perform cutting edge science in the field of gene
and cellular therapy and to promote the translation of basic discoveries into therapeutic advances.
The research projects span from basic studies aiming to:
- identify the genetic bases of inherited diseases;
- develop new gene transfer technologies for more efficient and safe genetic modification of target cells,
- establish procedures for isolation, gene transfer and transplantation of stem cells;
- modulate immune response to gene and cell products to improve efficacy and stability of the therapy, to
pre-clinical studies testing the experimental therapeutic strategies in disease models and eventually to the establishment of clinical trials of the new therapies in human
patients.
The genetic diseases, which are presently under investigation (from those in advanced clinical experimentation to those in early pre-clinical development) include
primary immunodeficiencies and some autoimmune disease, leukodystrophies and other lysosomal storage disorders, thalassemias, type I diabetes, hemophiliac. Furthermore,
we have established a
Pediatric Clinical Research Unit that focuses on the diagnosis, treatment and follow up of patients with primary immunodeficiencies, hematologic and
metabolic disorders, including those enrolled in the gene therapy trials. The TIGET clinical trial for a severe form of primary immunodeficiency (ADA-SCID) has provided the
most successful demonstration today that gene transfer into human hematopoietic stem cells can result in long-term correction of disease with an excellent safety record.
