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Bubble Babies: a new gene therapy

09 Oct 2014 | By Inserm (Newsroom) | Uncategorized

9 children with X-linked severe combined immunodeficiency have received a new form of gene therapy. Alain Fischer, Marina Cavazzana-Calvo and Salima Hacein-Bey-Abina, together with their teams at Inserm Unit 1163 and AP-HP, have just published these results in the New England Journal of Medicine.

In 1999, these researchers pioneered the treatment by gene therapy of children with a disease that left them without any immune defences. However, some of these children developed leukaemia after therapy. Following the development of safer therapeutic vectors, it was possible to start a new clinical trial in 2010.

4 years later, the results obtained and described in this scientific article are encouraging. 7 children treated using this method are now in good health.

For more information, see the press release “Les “bébés-bulle” ont 11 ans: L’efficacité de la thérapie génique est démontrée (“Bubble babies” are 11 years old: the efficacy of gene therapy is demonstrated),” published in July 2010

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