Our research

Rett Syndrome has a single genetic cause and is one of the only neurological disorders in the world to demonstrate dramatic symptom reversal in the lab.

Reverse Rett was founded in 2010 to compound efforts to accelerate the delivery of treatments and a cure for Rett Syndrome and related MECP2 disorders.

Since we launched, we've delivered over £5 million to Rett Syndrome research working in partnership with the US based Rett Syndrome Research Trust (RSRT) towards our common goal.

This research has made great strides in understanding the underlying cause of Rett Syndrome, finding ways to treat symptoms and figuring out how to reverse the condition completely with the help of some of the world's best scientists.

But what causes Rett Syndrome and why are we so sure that treatments and even a cure are really possible?

Rett Syndrome is most often caused by mutations in a gene called MECP2, on the X chromosome. 

This gene makes a protein, also called MeCP2 (but written differently), which is necessary for normal brain function. We know from the reversal experiments of 2007, that when this protein is replaced at adequate levels in mice, the symptoms of Rett Syndrome go away.

It wasn't until 2013, that researchers found a tangible way to translate those original reversal experiments into something that could potentially work in a human with Rett Syndrome; gene therapy.

RSRT immediately launched the Gene Therapy Consortium, a group of four elite labs, bringing together their unique area of expertise in order to troubleshoot all the problems inherent with translating this complex lab work, into a potentially life-changing therapy for people living with Rett Syndrome.

Here is the Roadmap to a Cure