Comment on the new study from Adrian Bird's research team at Edinburgh University (Science: Guy et al 2007)

Alison Kerr OBE MD February 2007

"The new research progress from Adrian Bird's department this month brings progress towards treatment for Rett syndrome to a new level.

The research showed that in a small number of mice, in which the gene MECP2 had been masked through the use of a special technique - and in which the clinical signs of Rett syndrome had already appeared - the return of the gene to normal activity leads to the disappearance of those abnormal clinical signs. Although mice are very different from humans they do need the activity of normal MECP2 and the lack of that gene's activity produces a condition that is remarkably similar to Rett syndrome in people.

This new research thus shows that the state of these mice due to the lack of the activity of MECP2 can be significantly improved when the gene is made active again. That provides a solid foundation for the hope that treatment may be developed that will fundamentally modify - perhaps cure - abnormalities that occur in Rett syndrome in people, even after the appearance of the full signs of the syndrome.

Of course much more research will have to be done in order to develop treatment that is effective and safe in Rett syndrome and to find how it can be delivered to the brain. New methods will have to be developed that are suitable for people with Rett syndrome and progress will depend on further research including working with animals whose brain is more similar to the human brain.