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A patient with severe sickle cell disease has undergone gene therapy developed at the CEA François-Jacob Institute. Two years after treatment, the remission of clinical signs was complete.
A clinical trial conducted by Prof. Philippe Leboulch from the François-Jacob Biology Institute, in collaboration with Prof. Marina Cavazzana from Hôpital Necker-Enfants malades, has led to the complete remission of sickle cell disease in an adolescent. Now 15 years old, this young boy had no compatible donor and presented particularly severe symptoms of the disease. He took part in the trial in September 2014. He now no longer requires any monthly transfusion or any other treatment to prevent sickle cell attacks. This complete remission of symptoms constitutes only a first step; the physicians will still have to wait a few more years before they can say it has been cured. The results of this clinical trial are yet another illustration of French expertise in the field of gene therapy.
The first phase of the clinical trial consisted of removing samples of hematopoietic stem cells, responsible for the production of all lineages of blood cells, from the bone marrow of the patient. A viral vector carrying a therapeutic gene already developed to treat β-thalassemia was introduced into these cells in order to correct them. Developed within the Innovative Therapies Department of the François-Jacob Institute, and produced on a mass scale by American company Bluebird Bio, this lentiviral vector is capable of transporting long segments of complex DNA. "We then reinjected the treated cells intravenously," said Prof. Leboulch. "Today, two years after administering the treatment, the patient produces more than 50% normal hemoglobin (instead of 20%, which would have been sufficient to treat the disease), with a complete inhibition of pathological sickle cell phenomena."
This result was the subject of a press release.
 A vector is a DNA or RNA molecule capable of self-replication (plasmid, cosmid, viral DNA) and in which foreign DNA is introduced. The vector is then used to insert this DNA into a target cell.
Gene Therapy in a Patient with Sickle Cell Disease | New England Journal of Medecine
CEA is a French government-funded technological research organisation in four main areas: low-carbon energies, defense and security, information technologies and health technologies. A prominent player in the European Research Area, it is involved in setting up collaborative projects with many partners around the world.