21 Apr 2015

New gene therapy success in a rare disease

Illustration for article: New gene therapy success in a rare disease
French teams from CIC Biothérapie (AP-HP/Inserm), from pediatric hematology department of Necker Hospital for Children (AP-HP), led by Marina Cavazzana, Salima Hacein Bey Albina and Alain Fischer and from Genethon led by Anne Galy (Genethon/Inserm UMR-S951), and English teams from UCL Institute of Child Health and Great Ormond Street Hospital in London led by Adrian Thrasher and Bobby Gaspar demonstrated the efficacy of gene therapy treatment for Wiskott-Aldrich Syndrome (WAS).

Six children that were treated and followed for at least 9 months
had their immune system restored and clinical condition improved. This work, which
was published today in the Journal of the American Medical Association (JAMA), was
carried out with support from the AFM-Telethon.
Syndrome (WAS).