San Raffaele Telethon Institute for Gene Therapy

Clinical research Unit


Head of Unit

Alessandro Aiuti



The Clinical Research Unit (CRU) was established in 1999 to create an infrastructure with dedicated staff and in house expertise for conducting clinical trials for genetic diseases. The overall goal of this Unit is the application of basic research discoveries and innovative gene and cell therapy approaches to the treatment of genetic diseases focusing on primary immunodeficiencies, genetic autoimmune diseases, hemoglobinopathies, and lysosomal storage disorders. It provides a suitable environment to translate the results from basic and clinical research into clinical practice and viceversa. The Unit was established through a dedicated grant of the Fondazione Telethon to SR-TIGET and is led by Alessandro Aiuti. During these years, the CRU has been recognized as a center of excellence for the treatment of pediatric and adult patients affected by genetic disorders. 


The Unit is composed by a Clinical Haematology Research Unit (CHRU) and a Pediatric Clinical Research Unit (PCRU) and a Clinical Trial Office, dedicated to the unit activity. All together the staff of the CRU has the required expertise for carrying in-house advanced therapies-based clinical trials according to GCP. The CRU acts in strict cooperation with the Pediatric Immuno‐Hematology Unit of IRCCS Ospedale San Raffaele and the San Raffaele Stem Cell Program. 

The CHRU and PCRU have dedicated spaces and personnel, including physician scientists, hematologists, pediatricians, resident fellows in Pediatrics, physiotherapist, psychologist, and nurses.

The use of standardized processes and SOP guarantees high quality of the clinical trials. The approach to the patients is holistic, taking charge of all the medical, nursing and psychological aspects thanks to a multidisciplinary team. The management of patients and their families takes into account the handling of emotional issues, the different cultural, social and economic backgrounds as well as the logistical problems related to the prolonged stay of patients and families in Milan

Research activity

Clinical research activities are focused on the implementation of experimental protocols based on gene or cell therapy for the treatment of genetic blood disorders such as ADA-SCID, Wiskott-Aldrich Syndrome, Metachromatic Leukodystrophy, Beta-thalassemia, Chronic granulomatous disease, Mucopolysaccharidosis Type I (Hurler).

Studies to improve the knowledge of genetic diseases include the natural history study for MLD, advanced diagnosis and natural history of primary immunodeficiencies and immunedysregulatory disorder, and immune reconstitution after hematopoietic stem cell transplantation for genetic disorders.

For more detailed information about research activity of Clinical research Unit click here.


The CRU and its cooperating Units obtained different accreditation, ensuring high quality standards for its activities:

  • ISO 9001 certification for Pediatric Immuno–Hematology Unit, Hematology and Bone Marrow Transplant Unit and SR-TIGET Clinical Trial office
  • AIFA self-certification for conduction of Fase I clinical Trial
  • JACIE certification for the Stem Cell Program European Blood and Marrow Transplantation group
  • Associazione Italiana Ematologia ed Oncologia Pediatrica (AIEOP)
  • Gruppo Italiano Trapianto Midollo Osseo (GITMO)
  • European Reference Network_Rare Immunodeficiency, autoinflammatory and autoimmune disease Network (ERN-RITA)

The Unit participates to the scientific activity of: Società Italiana di Pediatria, Società Italiana di Ematologia, American Society of Hematology (ASH), American Society of Gene Therapy (ASGCT), European Society of Cell and Gene Therapy (ESGCT).