The San Raffaele Telethon Institute for Gene Therapy (TIGET)
Director: Luigi Naldini
The San Raffaele Telethon Institute for Gene Therapy (TIGET), directed by Luigi Naldini, is a joint-venture between the San Raffaele Scientific Institute and the Telethon Foundation. The mission of the Institute is to perform innovative research on gene transfer and cell transplantation and translate its results into successful clinical application of gene and cell therapy for genetic diseases.
The research activities of the Institute span from basic to early phase clinical studies aiming to:
i) identify the genetic bases and the pathophysiological processes of the inherited diseases under study, which include primary immunodeficiencies, blood diseases, inherited leukodystrophies and other lysosomal storage diseases;
ii) develop new gene transfer and editing technologies for more efficient and safe genetic correction of diseases using ex vivo and in vivo strategies;
iii) characterize the biological properties of hematopoietic and neural stem cells and establish procedures for their ex vivo manipulation and transplantation;
iv) investigate cell types mediating innate and adaptive immunity and modulate immune response to gene and cell products to improve efficacy and stability of the therapy;
v) test the new therapeutic strategies in preclinical disease models instrumental to design clinical trials;
vi) exploit the technologies and models developed above to design new gene therapy strategies for cancer.
All these goals are fulfilled within the TIGET Experimental Research Units;
vii) assess the efficacy and safety of the novel therapeutic strategies in patients within early phase clinical trials. This clinical activity is conducted within the TIGET Clinical Research Unit (CRU; Clinical Research Coordinator: Alessandro Aiuti), which is devoted to the diagnosis, treatment and follow-up of patients with primary immunodeficiencies, hematologic and metabolic diseases, and comprises the TIGET Clinical Trial Office (TCTO). The TIGET Clinical Research Unit works in close collaboration with the Pediatric Immuno-Hematology Unit (directed by Alessandro Aiuti) and the Hematology and Bone Marrow Transplantation Unit (directed by Fabio Ciceri) of the San Raffaele Hospital.
Moreover, TIGET includes the Vector Integration Core, aimed at monitoring and profiling vector integration genome-wide in preclinical and clinical studies, and a GLP test facility, aimed at conducting preclinical studies of gene and cell therapy according to GLP (Good Laboratory Practice) guidelines.
The gene and cell therapy products used in the current clinical trials are produced in the GMP (Good Manufacturing Practice) facility of MolMed S.p.A.
Overall, this research environment provides world-recognized leading scientific expertise in gene transfer and editing technologies, genetic modification of stem cells, preclinical models of stem cells transplantation, as well as in the translational, regulatory and clinical issues associated with testing novel cell and gene therapies for immune, hematological and lysosomal storage diseases.
The Institute is directed by Luigi Naldini since 2008. The Director is supported by a Clinical Research Coordinator (Alessandro Aiuti), 6 Heads of Unit, 3 Group Leaders, 5 Project Leaders, and a total of 150 people including fellows, PhD students, post-doctoral fellows and administrative staff, comprising a planning and cost controller, a scientific officer, a regulatory affairs manager, alliance managers and a secretariat.
Applications now open for the International PhD Course in Molecular Medicine a.y. 2015-2016 at UniSR.
We are looking for highly motivated scientists to be enrolled as Postdoctoral Fellows in the experimental and clinical research groups of TIGET. Interested candidates are invited to go through the list of TIGET research groups presented below and to send their application, including CV and reference letters, directly to the specific research group of interest.
Experimental research and clinical research groups
- Luigi Naldini, Director, Head of Unit
- Alessandro Aiuti, Clinical Research Coordinator, Head of Unit
- Alessandra Biffi, Head of Unit
- Giuliana Ferrari, Head of Unit
- Angela Gritti, Head of Unit
- Anna Villa, Head of Unit
- Bernhard Gentner, Group Leader
- Silvia Gregori, Group Leader
- Eugenio Montini, Group Leader
- Anna Kajaste-Rudnitski, Project Leader