The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget)

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Director: Luigi Naldini
Deputy Director: Alessandro Aiuti

The San Raffaele Telethon Institute for Gene Therapy (SR-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 SR-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 SR-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 SR-Tiget Clinical Trial Office (TCTO). The CRU 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, SR-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.

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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 Deputy Director and Clinical Research Coordinator, Alessandro Aiuti. The experimental and clinical research groups present in SR-Tiget are listed below. The staff consists of a total of 180 people, including Heads of Unit, Group Leaders, Project Leaders, post-doctoral fellows, PhD students, fellows and administrative staff, comprising a planning and cost controller, a scientific officer, a regulatory affairs manager, alliance managers and a secretariat.


Job opportunities

  • Scientific Officer
    position open at SR-Tiget. More info here.
  • Alliance Manager & Regulatory Affairs
    position open at SR-Tiget. More info here.
  • Technician Position
    open in Alessandro Aiuti’s Lab – Pathogenesis and therapy of primary immunodeficiencies. More info here.
  • Postdoctoral Fellow
    positions open in Renato Ostuni’s Lab – Genomics of the innate immune system. More info here.
  • Postdoctoral Fellow
    position open in Silvia Gregori’s Lab – Mechanisms of Peripheral Tolerance. More info here.
  • Postdoctoral Fellow, Research Fellow and PhD candidate
    positions open in Andrea Ditadi’s Lab – Human hematopoietic development and disease modeling. More info here.
  • Postdoctoral Fellows
    We are looking for highly motivated scientists to be enrolled as Postdoctoral Fellows in the experimental and clinical research groups of SR-Tiget. Interested candidates are invited to go through the list of SR-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

Gene transfer technologies and new gene therapy strategies

Angiogenesis and tumor targeting

Alessandro Aiuti Deputy Director, Clinical Research Coordinator, Head of Unit

Pathogenesis and therapy of primary immunodeficiencies

Clinical Research Unit (CRU)

Alessandra Biffi Head of Unit

Hematopoietic stem cell gene therapy for lysosomal storage disorders

Giuliana Ferrari Head of Unit

Gene transfer into stem cells

Angela Gritti Head of Unit

Gene/neural stem cell therapy for lysosomal storage diseases

Eugenio Montini Head of Unit

Safety of gene therapy and insertional mutagenesis research

Anna Villa Head of Unit

Pathogenesis and treatment of immune and bone diseases

Bernhard Gentner Group Leader

Translational stem cell and leukemia research

Silvia Gregori Group Leader

Mechanisms of peripheral tolerance

Angelo Lombardo Group Leader

Epigenetic regulation and targeted genome editing

Marita Bosticardo SR-Tiget Group Leader

Thymus biology and engineering

Raffaella Di Micco SR-Tiget Group Leader

Senescence in stem cell aging, differentiation and cancer

Andrea Ditadi SR-Tiget Group Leader

Human hematopoietic development and disease modeling unit

Anna Kajaste-Rudnitski SR-Tiget Group Leader

Retrovirus-host interactions and innate immunity

Renato Ostuni SR-Tiget Group Leader

Genomics of the innate immune system