She is a Full Professor of Hematology at Université Paris Cité, Head of the Biotherapy Department at Necker Enfants Malades Hospital (Assistance Publique – Hôpitaux de Paris), and Coordinator of the Clinical Investigation Center in Biotherapy at the Imagine Institute in Paris, France.
Her basic, translational, and clinical research is dedicated to the development and implementation of innovative therapies for genetic diseases, with a focus on gene and cell therapies using genetically engineered autologous hematopoietic stem and progenitor cells (HSPCs).
Her work supports the development of transformative therapies that overcome the limitations of the lack of HLA-identical family donors, advancing personalized medicine through genetic engineering approaches.
She has also contributed to the discovery of pathogenic genetic variants involved in rare hematopoietic diseases, leveraging this knowledge to develop novel therapeutic strategies.
She is a member of the U.S. National Academy of Medicine and the French Academy of Medicine.
She is affiliated with the Biotherapy Department at Necker Enfants Malades Hospital, the Clinical Investigation Center in Biotherapy (INSERM), the Imagine Institute, and Université Paris Cité.
She currently serves as Full Professor of Hematology at Université Paris Cité and Head of the Biotherapy Department at Necker Enfants Malades Hospital (AP-HP).
