About this Talk
In this talk, Jean-Christophe Roux presents the important points concerning gene therapy in Rett syndrome. He shows that this technique is probably one of the most promising to correct the pathology completely. Nevertheless, there are several difficulties such as the fact that only half of the cells of the girls must be treated, the possibility of overdosing of the Mecp2 gene, the route of administration of the viral vectors and finally the side effects on the liver. Despite these difficulties, several teams have already obtained interesting results are making rapid progress. In addition, the first clinical trials are being proposed by several organizations.