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10 March 2017


French doctors have reported the first successful patient treatment with a gene therapy for sickle cell disease.

According to the report, the 13-year-old patient still has no need of transfusion treatments 15 months after treatment.

The patient had fully resumed school and no longer suffers from vaso-occlusive crises, the doctors reported in The New England Journal of Medicine.

The treatment used a lentiviral vector developed by Professor Philippe Leboulch, of University Paris-Sud, France, and carrying long complex DNA segments.

The vector carried a therapeutic gene previously developled to treat beta-thalassaemia and correcting the gene encoding beta-globin. This was introduced into the patient's haematopoietic stem cells.

Professor Leboulch said: 'We note that the expression of the therapeutic protein from the vector, highly inhibiting pathological sickling, is remarkably high and effective.'

Fellow researcher Professor Marina Cavazzana, of the University Paris-Descartes, said: 'We hope, with this gene therapy approach, to develop future clinical trials and include a significant number of patients with sickle cell disease, in the Ile-de-France and in the national territory.'

Source: Gene Therapy in a Patient with Sickle Cell Disease NEJM 2 March 2017;

Link: http://www.nejm.org/doi/full/10.1056/NEJMoa1609677


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