Adrian Bird shows Rett can be reversed in mice, a huge moment that paves the way for Rett gene therapy research in humans.
In 2007, with the successful reversal of Rett in the lab, we entered a new era for research in which we knew a cure was possible. We are now entering a final era, in which trials are beginning and we are building the clinical capacity to deliver effective treatments.
Below, you can see a timeline of key milestones that show progress in the field since the reversal experiments of 2007 and in the work of Reverse Rett which was launched in 2010.
Adrian Bird shows Rett can be reversed in mice, a huge moment that paves the way for Rett gene therapy research in humans.
Reverse Rett is founded by five parents from the home of now CEO, Rachael Stevenson.
Launch of RSRT’s international MECP2 Consortium to combine global efforts to find a tangible way to reverse Rett in humans.
RSRT launch international Gene Therapy Consortium to compound efforts to drive Gene Therapy for Rett into human trials.
Gene therapy shown to reverse the symptoms of Rett Syndrome in mice.
Reverse Rett start UK Rett Syndrome Patient Registry in anticipation of human clinical trials.
Reverse Rett begin seed-funding a pioneering team at King’s College London, working to develop new outcome measures for patients with Rett Syndrome under Professor Paramala Santosh.
UK’s first clinical trial of a potential treatment for one of the symptoms of Rett Syndrome begins under the leadership of Professor Paramala Santosh at King’s College London.
RSRT/AveXis announcement of first human clinical trial for gene therapy in Rett-another major milestone that demonstrates a cure is within our reach.
2nd UK clinical trial for a potential treatment (cannabidiol) launched at three UK trial sites
We’re working to bring about better days for children and adults living with Rett Syndrome today by funding international laboratory research focused on addressing the underlying cause of the disease and UK clinical research, which will enable children and adults with Rett to lead longer, healthier lives until treatments and a cure become available.
We use a rigorous application process, called peer review, to help us decide which UK clinical research to fund.
People with Rett Syndrome are at the heart of everything we do. Through research, we aim to make a positive impact on their lives. Reverse Rett is a full member of the Association of Medical Research Charities (AMRC) and are the leading funder of Rett Syndrome research in the UK.
I was first introduced to Rett Syndrome when I received an out of the blue phone call from Adrian Bird, who first discovered MeCP2 protein, requesting some help with a set of specific experiments. Following this successful collaboration, Rett Syndrome research has become a primary focus of my lab. While traditional drug approaches will likely be restricted to correcting specific aspects of what goes wrong in Rett, it is conceivable that Gene Therapy can correct the cause of Rett at it’s very source and thus provide a profound recovery of function.
