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.