Marina Cavazzana, MD, PhD

Marina Cavazzana is a pediatrician, Professor of Haematology since 2000, Head of the Biotherapy Department at Hospital Necker, University Paris Descartes. She is the Director of the Inserm / Assistance Publique – Hôpitaux de Paris GHU Ouest Biotherapy Clinical Investigation Center and leads a research Laboratory at Imagine Institute. She studied medicine in Padua, Italy and received Doctorate of Medicine in 1983, her certification in Paediatrics in 1987 and a PhD in Life Sciences in 1993 (University Paris VII). Dr. Marina Cavazzana has dedicated her time and research in investigating innovative protocol with a particular intervention gene therapy, focusing on sickle cell disease. Her main research and clinical interests are development of the immune system, genetic diseases of the haematopoietic system and cell and gene therapy. Her group studies the means to improve the clinical results of hematopoietic stem cell transplantation, crossing HLA-barriers, and the differentiation of mouse and human stem cells towards lymphocyte lineages. She has initiated several clinical trials based on the use of ex vivo gene modified cells to treat patients with inherited disorders, the preliminary clinical results of which are encouraging. She is the author or co-author of one patent and of more than 270 publications in peer-reviewed journals.   Representative Publications: Gene Therapy in a Patient with Sickle Cell Disease Family cord blood banking for sickle cell disease: a twenty-year experience in two dedicated public cord blood banks Arterio-venous fistula for automated red blood cells exchange in patients with sickle cell disease: Complications and outcomes Cardiac iron overload in chronically transfused patients with thalassemia, sickle cell anemia, or myelodysplastic syndrome Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel Online profile
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