The San Raffaele Telethon Institute for Gene Therapy (TIGET)

Versione italiana

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 established 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 activity of the Institute spans from basic to pre-clinical studies aiming to:

i)   identify the genetic bases and the pathophysiological processes of the inherited diseases under study, which include primary immunodeficiencies and autoimmune disorders, thalassemia, inherited leukodystrophies and other lysosomal storage disorders;

ii)   develop new gene transfer and editing technologies for more efficient and safe genetic correction of disease ex vivo and in vivo;

iii)  characterize the biological properties of hematopoietic and neural stem cells and establish procedures for 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 pre-clinical disease models instrumental to design clinical trials (same link used above for genetic bases and the pathophysiological processes);

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 Pediatric Clinical Research Unit (P-CRU; Clinical Activities Coordinator Alessandro Aiuti) that is devoted to the diagnosis, treatment and follow-up of patients with primary immunodeficiencies, hematologic and metabolic disorders, in close collaboration with the Pediatric Immunohematology Unit, directed by Maria Grazia Roncarolo, the Bone Marrow Transplantation Unit, directed by Fabio Ciceri, and the GMP Facility MolMed SpA.

Moreover, TIGET includes the Vector Integration Core, aimed at studying vector integration and its impact on gene transcript structure and expression, and a GLP Center (certification pending), aimed at conducting pre-clinical studies according to Good Laboratory Practice (GLP) guidelines.

TIGET activity transversally addresses three major disease settings: Primary Immunodeficiencies, Lysosomal Storage Disease and Blood Disorders.

Overall, this research environment provides world-recognized leading scientific expertise in vector design and assays, genetic modification of HSC, pre-clinical models of HSC activity as well as in the translational, regulatory and clinical issues associated with testing novel therapies for immune and hematological diseases.

 

TIGET Structure

The Institute is directed by Luigi Naldini since 2006; the Director is supported by a Research Activities Coordinator (Giuliana Ferrari), a Clinical Activities Coordinator (Alessandro Aiuti), four Head of Units, five Group Leaders and five Project Leaders, with a total of 130 personnel including Post-Docs, PhD Students, Fellows and administrative staff. The administrative staff includes Michela Gabaldo, the Alliance Manager of the Telethon-GSK jointed project, Massimiliano Bollina (accounting), and, with secretariat function, Teresa Cascella, Luisella Meroni, Giuliana Tomaselli, Luca Libelli and Giulia Gagliasso. Gioconda Velasques and Samih El Hossary are the support staff.

 

Research units/clinic research groups