Since Reverse Rett was founded in 2010, we’ve delivered over £6 million to international Rett syndrome and MDS research through our partnership with the US based, Rett Syndrome Research Trust (RSRT).
RSRT are responsible for peer review and monitoring of Rett Syndrome and MECP2 Duplication projects. These projects primarily aim to transform the lives of children and adults with these disorders.
The focus of our UK clinical work is to ensure that as treatments and a cure for Rett Syndrome become viable, they are accessible to patients in the UK.
CIPP Rett Centre is a Rett Syndrome clinical research and treatment centre providing holistic care for affected children and adults and generating critical research outputs at the same time.
The team are the first in the world to have developed a digital monitoring platform for Rett Syndrome which tracks patients’ symptoms on an ongoing basis over time; the CIPP Rett Database.
The learning from this has already enabled the research team to publish four papers and critically, to identify physiological outcome measures which could be used to measure efficacy in future human clinical trials.
To join the CIPP Rett Database or for more information about a referral to CIPP Rett Centre, please email CPMRS@slam.nhs.uk or CPMRS@reverserett.org.uk
Rett Syndrome has already been reversed in the lab, but before it can be replicated in people, researchers need to be sure that therapies are safe and effective. Too much MeCP2 protein can be as bad as too little; the level needs to be ‘just right.’ For many years Reverse Rett has funded work of the Gene Therapy Consortium at RSRT which led directly to the development of three commercial gene therapy products projected to launch human clinical trials for Rett Syndrome in late 2021.
While traditional drug approaches will likely be restricted to correcting specific aspects of what goes wrong in Rett Syndrome, 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.”
Dr Stuart Cobb, Simons Fellow and Reader in Neuroscience at the Patrick Wild Centre and Centre for Discovery Brain Sciences, University of Edinburgh.