Gene and cell therapy
The program, which cuts across the four curricula, aims to train young scientists in the study of genetic diseases and in the development of new gene and cell therapy strategies, with a specific focus on the translational aspects of research, in view of future clinical applications. The development of such therapies requires a complex path, which passes through the identification of the mutated genes and the physiopathological mechanisms underlying the genetic diseases under investigation, the design of new therapeutic strategies and their validation in vitro and in animal models of disease, up to clinical trials in patients.
Specifically, the program will offer a wide spectrum of educational activities, including strategies of gene and cell therapy, gene transfer and genome editing technologies, procedures for the manipulation and transplantation of stem cells, basic normative and regulatory requirements of preclinical studies and clinical trials. For a list of projects relating to Gene and cell therapy program click here.
The program is supported by the Telethon Foundation, in line with its mission to support biomedical research on genetic diseases, and sees the active contribution of the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), an international reference center for gene and cell therapy.
Human hematopoietic development and disease modeling
Supervisor: Andrea Ditadi
Mechanisms of peripheral tolerance
Supervisor: Silvia Gregori
Gene and neural stem cell therapy for lysosomal storage diseases
Supervisor: Angela Gritti
Genomics of the innate immune system
Supervisor: Renato Ostuni
Pathogenesis and therapy of primary immunodeficiencies
Supervisor: Alessandro Aiuti | Alessandra Mortellaro | Maria Ester Bernardo