Department of Paediatric Immunohaematology @ Ospedale San Raffaele

The Paediatric Immunohematology Unit is a centre of excellence for the treatment of genetic blood disorders by gene and cell therapy. The Unit deals with the diagnosis and treatment of children affected by haematological, immunological and metabolic diseases with neurological involvement, with particular regard to genetic diseases. The centre is composed of a highly specialized team with a long experience in the diagnosis, research and treatment of patients suffering from genetic diseases. The Unit is the Reference Centre for the diagnosis of Primary Immunodeficiency Disease (PID) and other paediatric haematological diseases.

The Unit actively collaborates with the San Raffaele’s Telethon Institute for Gene Therapy (SR-Tiget) for the clinical application of the experimental protocols of gene and cell therapy and is part of the Stem Cells Programme for the transplantation of hematopoietic stem cells. Clinical and research activity is documented by a large number of publications in national and international scientific journals.

The Unit’s activity is divided into the following specialized branches:

  • Paediatric immunology
    • Primitive immunodeficiency
    • Autoimmune diseases
  • Paediatric haematology
    • platelet diseases
    • Anaemias
    • Neutropenia
  • Neuropediatric metabolic diseases

Our excellence in numbers


gene therapy/transplantations

Notable advanced technological equipment

The Unit’s diagnostic and research laboratories equipment allows the most precise diagnosis of immunological and haematological defects, i.e. genetic investigations (next generation sequencing, in collaboration with the laboratory of molecular clinical biology), flow cytofluorimetry, functional tests.

The SR-Tiget Clinical Lab is a certified laboratory, in which tests required for clinical trial endpoints are performed following GCLP requirements. The SR-Tiget Clinical Lab can perform tests for all phases Clinical Trials, including phase I trials.

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Meet our team

Head physician

Alessandro Aiuti

Alessandro Aiuti
Ospedale San Raffaele

The San Raffaele Research Hospital is a highly specialized multi-disciplinary medical center of world importance, which embodies the integrity of the treatment, clinical research and teaching activities. San Raffaele provides treatment in all existing medical fields, which allows this center to take care of the most complex patient cases. The Hospital is recognized as IRCCS (Scientific Institute for Research and Healthcare) for its research in molecular medicine and innovative treatments application, such as the gene therapy.

The hospital is a multi-specialty center with more than 50 clinical specialties available and with over 1600 beds; it is accredited by the Italian National Health System to provide care to both public and private, Italian and international patients. In 2018 the San Raffaele Hospital performed over 51,000 patient admissions, 72,000 emergency room encounters and delivered over 1.5 million outpatient medical services. It is widely regarded as the most celebrated hospital in the country and among the most reputable medical centers in Europe. San Raffaele’s wards and operating theatres are equipped with the most advanced machines, such as PET, PET/CT, Gamma Knife, Cyber Knife, Rapid Arc, Tomotherapy. San Raffaele’s Radiotherapy Unit was the first place in Europe to install Tomotherapy in 2004. Nowadays this Unit confirms to be one of the most experienced worldwide; it has developed numerous protocols for cancer treatment of the prostate, breast, central nervous system, brain, liver, pancreas, gastrointestinal tract, lungs, and others. The Oncological Board of the Hospital consisting of diagnosticians, clinical oncologists, immunologists, surgeons and radiotherapists, hold weekly meetings to discuss oncological patients’ pathways in a multidisciplinary manner.

Beside its Cancer Center, San Raffaele is well-known for its medical know-how in cardiology and cardiac surgery, neurology and neurosurgery, gastroenterology and gastrointestinal tract surgery, liver surgery, pancreas surgery, transplant medicine, gynecology and obstetrics, urology, dermatology, ophthalmology, immunology, rheumatology and pediatrics.

San Raffaele is a pioneer in the field of gene medicine and treatment of rare immunologic and genetic diseases. San Raffaele Scientific Institute is where the first in the world gene therapy with stem cells was created, named Strimvelis, for patients affected by ADA-SCID syndrome.

The Research at the San Raffaele Hospital focuses on integrating basic, translational and clinical research to provide the most advanced care to the patients. It counts on over 1,500 scientists, working in state-of-the-art facilities covering a surface of 130,000 square meters, and have produced over 1,446 scientific publications in 2018. Research at San Raffaele aims at understanding the mechanisms underlying a variety of important human diseases and at identifying new targets and new therapeutic strategies to treat them.

The San Raffaele Hospital hosts the Università Vita-Salute San Raffaele, a fully private university with a complete Medical School (including specialty courses and residencies), Nursing School, a Psychology graduate and post-graduate programs, and Allied Health Professions School. The mission of the Vita-Salute San Raffaele University is to answer the question "Quid est homo?" in the belief that the human being is a biological, psychological and spiritual unicum. Since 2010 the San Raffaele Hospital also hosts the International MD Program, the only graduate program accredited to license Medical Doctors in both Europe and North America.


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