Dr. Annie Harroche et Dr. Delphine Borgel
Gene therapy in severe haemophilia A (factor VIII deficiency <1%) is a very promising future therapy since gene correction directly adjusts factor VIII levels. In addition, a correction of a few % of the circulating factor levels makes it possible to transform a severe phenotype into a minor phenotype, with little haemorrhage. A gene therapy project in this pathology is currently being developed in close cooperation between the haemophilia treatment center of Necker Hospital which follows a cohort of patients with severe haemophilia A; the hemostasis laboratory of the Necker Hospital and the team of the Imagine Institute of Prof. Marina Cavazzana. The objective of this project is to develop a clinical and biological surveillance (anti-adenovirus serology, T lymphocyte immune response, transgenic factor VIII specific activity assay, anti-factor VIII specific antibody search) in order to follow the evolution of the cohort of patients in the development of gene therapy in severe haemophilia A.
Hôpital Necker Enfants Malades
149, Rue de Sèvres