Marina Cavazzana

Professor in Haematology, Head of the Biotherapy Department, Director of the Clinical Investigation Center for Innovative Therapies | Isabelle André

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Marina Cavazzana

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Marina Cavazzana is a pediatrician, Professor of Hematology since 2000, Director of the Department of Biotherapy at Necker Hospital, Paris Descartes University. She is the Director of the Inserm / Assistance Publique - Hôpitaux de Paris GHU Ouest Biotherapy Clinical Investigation Center and is a member of the research Human Lymphohematopoiesis laboratory at Imagine Institute.

Her main research and clinical interests are the development of the hematopoietic immune system, and cell and gene therapy for inherited and acquired disease of the hematopoietic system. 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 hematopoietic stem cells to treat patients with inherited disorders, the preliminary clinical results of which are encouraging. She is the author or co-author of several patents and of more than 250 publications in peer-reviewed journals and was awarded 2 ERC (2011 and 2016) and one state funding from the French National Research Agency (ANR) Investments for the Future programme (PIA) (Recherche Hospitalo-Universitaire en santé) (2019).

Her work was rewarded by the American Society of Hematology (Award on Clinical Research in Gene Therapy in 1999), by the French Academy of Sciences (Special Medical Award in 2000 and Jean-Pierre Lecocq Award on Gene Therapy in 2004). She was awarded the title of Officier de l'Ordre National de la Légion d'honneur in 2011, and given the Irène Joliot Curie 2012 award "Scientific Women of the Year" (Science Academy and French Ministry of Education and Research). She was also awarded with the French National Academy of Medicine in 2016 and the 2017 Ernest Beutler Lecture and Prize for Clinical Science (American Society of Hematology). She has also been elected as an International member of the National Academy of Medicine in 2019.

Abstract 04

Research: a scientific adventure

Our goal: to better understand genetic diseases to better treat them.

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