Certain key things are known about what causes Rett, which is a huge advantage in terms of trying to find treatments and a cure. We know that: It is a single gene disorder. It has been widely demonstrated to be reversible when the missing protein is replaced
Again, this is rather a complex scientific notion for most of us (well, me!), but in short the idea is to identify other genes which, when mutated, somehow protect an individual from a MeCP2 mutation. There are people who have common MECP2 mutations and normal X
There are several different approaches in terms of research: putting the protein back: this approach aims to recreate the successful reversal experiments on mice, from 2007, which showed that putting the MeCP2 protein back reverses the symptoms of Rett. improving symptoms: many of the symptoms of Rett could