The scientific coordinator

The scientific coordination of the network is provided by Prof. Marina Cavazzana.

In 1999, she set up at the Necker Hospital, with Prof. Alain Fischer and Salima Hacein Bey Abina, a first gene therapy protocol for children with severe immunodeficiency, a rare genetic disease. This treatment consisted of harvesting hemopoietic stem cells (the stem cells responsible for all blood cell lines, including cells of the immune system) in patients, and then, inserting into these cells a “drug” gene with the help of a retroviral vector, modifying the defective gene. The modified cells, after checking their viability, were then reinjected into the children, without risk of rejection, unlike a bone marrow transplant treatment.