Imagine the symptoms of autism, cerebral palsy, Parkinson's, epilepsy, and anxiety disorders…all in one little girl.

Now imagine

these symptoms vanishing...one by one.

The Reversal

In 2007, a British scientist called Dr Adrian Bird successfully reversed the symptoms of Rett Syndrome in adult mice.

He did this by restoring protein levels in the faulty MECP2 gene. The results were as astonishing as they were unexpected. Until this point it had been assumed that the disorder was permanent and irreversible. Suddenly, there was proof of concept that girls destined to be profoundly disabled for the rest of their lives could be cured. Now the race was on to find a way of delivering the treatment to girls.

Recent studies show that, contrary to previous assumptions, sufferers of Rett Syndrome are NOT brain-damaged.

The neurons are intact. It is the synapses which are not firing because of the MECP2 gene which acts as a kind of master switch. This means that cognition and learning are retrievable. It also confirms what parents of children with Rett Syndrome have long known - that sufferers understand far more than they can express. The problem is one of communication. These girls are locked inside their bodies, desperate to communicate. They are trapped inside bodies that no longer work.

Curing Rett would not only benefit hundreds of thousands of girls but would also have huge implications for other neurological disorders like Autism, Parkinson’s and Cerebral Palsy. Rett Syndrome is the gateway to curing these conditions.

There is no government or lottery funding for research into Rett Syndrome.

Scientists rely wholly on private donations, despite the huge and wide-reaching promise of their research.

There will be no cure without more funding.

All the girls suffering are in a race against time.

How we get to a cure

Fast forward to 2022 and the results of that experiment are starting to bear fruit.

Taysha Gene Therapies have received approval for the first-ever gene therapy trial into women with Rett Syndrome.

It is just one of many approaches to curing this disease, alongside Gene Editing, MECP2 Reactivation and RNA Editing.

There are now 8 biopharmaceutical companies pursuing gene therapies for Rett Syndrome. All of them have been funded by Reverse Rett and its parent  charity Rett Syndrome Research Trust.

Please click here for more details on our strategy to cure Rett and the latest scientific advancements.

CURE 360

If you would like to find out more, a longer video is available here:

Together We Cure

Results of gene therapy in mice

These mice are male "Rett" mice. The mouse moving around was given gene therapy treatment and received healthy MECP2 gene. The immobile mouse did not receive treatment. The video was taken 4 weeks after treatment. This work was funded by RSRT.