Maria Grazia Roncarolo
Pediatric Clinical Research Unit
A Pediatric Clinical Research Unit (PCRU) has been established since 1999 at HSR-TIGET for the translation of basic research into the treatment of genetic diseases. The overall goal of this Unit is the application of basic research discoveries 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 HSR-TIGET. During these years, the PCRU has been recognized as a center of excellence for the treatment of patients affected by genetic diseases of the blood and the nervous systems.
The Unit is led by Prof Maria Grazia Roncarolo and operates in cooperation with the Pediatric Immuno-Hematology and Bone Marrow Transplantation Unit of San Raffaele Hospital with dedicated spaces and personnel of physician scientists, clinicians, nurses, and technical staff. The clinical activity of the Unit is mainly focused on hematopoietic stem cell transplantation and gene therapy for genetic disorders. In 2004, the San Raffaele Hospital and the PCRU started a cooperation with the Mediterranean Institute of Hematology (IME) for the treatment of patients affected by hemoglobinopathies. Our Institute is participating to this network with the aim to offer cure, research and training for patients, doctors and nurses from the underdeveloped Mediterranean countries.
The Pediatric Bone Marrow Transplant Unit comprises 6 inpatients rooms (one with laminar flow) and 6 Day-Hospital beds, next to the adult BMT Unit. Starting from 2009 a dedicated BMT pediatric area including 10 inpatients rooms will be in place in a new building. Patients who are not in need for special isolation are hospitalized in the Division of Pediatrics, which hosts 50 beds, 40 for children and 10 for newborns. The outpatient activities (evaluation, diagnosis, follow up) are carried out in three different outpatient clinics: one for immunological disorders, one for anemia/thalassemia, and one for rare neurological disorders. The Unit had obtained ISO certification and is approved from the Italian Pediatric Hematology-Oncology Association (AIEOP). The production of gene transfer vector and the procedures of cell manipulation and gene transfer for clinical protocols are currently performed by an authorized biotechnology company (MolMed S.p.A.) according to cGMP.
The Unit is led by Prof Maria Grazia Roncarolo and operates in cooperation with the Pediatric Immuno-Hematology and Bone Marrow Transplantation Unit of San Raffaele Hospital with dedicated spaces and personnel of physician scientists, clinicians, nurses, and technical staff. The clinical activity of the Unit is mainly focused on hematopoietic stem cell transplantation and gene therapy for genetic disorders. In 2004, the San Raffaele Hospital and the PCRU started a cooperation with the Mediterranean Institute of Hematology (IME) for the treatment of patients affected by hemoglobinopathies. Our Institute is participating to this network with the aim to offer cure, research and training for patients, doctors and nurses from the underdeveloped Mediterranean countries.
The Pediatric Bone Marrow Transplant Unit comprises 6 inpatients rooms (one with laminar flow) and 6 Day-Hospital beds, next to the adult BMT Unit. Starting from 2009 a dedicated BMT pediatric area including 10 inpatients rooms will be in place in a new building. Patients who are not in need for special isolation are hospitalized in the Division of Pediatrics, which hosts 50 beds, 40 for children and 10 for newborns. The outpatient activities (evaluation, diagnosis, follow up) are carried out in three different outpatient clinics: one for immunological disorders, one for anemia/thalassemia, and one for rare neurological disorders. The Unit had obtained ISO certification and is approved from the Italian Pediatric Hematology-Oncology Association (AIEOP). The production of gene transfer vector and the procedures of cell manipulation and gene transfer for clinical protocols are currently performed by an authorized biotechnology company (MolMed S.p.A.) according to cGMP.
