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NATURE: Gene-therapy boost

NATURE: Gene-therapy boost

Gene-therapy boost A gene-therapy treatment for patients with the blood-clotting disorder haemophilia B has scored its first unequivocal success, scientists at University College London reported at the American Society of Hematology meeting in San Diego, California, on 11 December (A. C. Nathwani et al. N. Engl. J. Med. http://doi.org/g8n; 2011). Haemophilia B is caused by mutations in the gene that codes for the factor IX protein; the treatment uses viruses to deliver a healthy version of the gene to patients' liver cells. In a previous trial, protein production dropped below therapeutic levels after two months (C. S. Manno et al. Nature Med. 12, 342–347; 2006). But in the latest trial, four out of six patients were still making the protein up to 18 months after one treatment, and did not need injections of blood-clotting factors.

http://www.nature.com/news/seven-days-9-15-december-2011-1.9644