Curing Rett

Mutations in a gene called MECP2 cause Rett Syndrome. Restoration of adequate levels of MECP2 has been shown to undo the damage caused by a mutated copy of the gene, demonstrating the powerful reach of MECP2‘s influence as symptom after symptom disappeared in fully mature mouse models of Rett Syndrome.  This astonishing breakthrough presents us with the urgent challenge of determining whether such results can be achieved in humans.

There are two primary approaches to reversing Rett Syndrome. The first is to understand the function of the MeCP2 protein and to design rational drugs to compensate for its deficit. The second approach is to identify the various outcomes of having an MeCP2 deficiency and screen for anything that ameliorates that outcome. In collaboration with RSRT; Rett Syndrome Research Trust UK has supported twelve different research projects focused on these solutions.

The table below shows the research projects we have supported since our launch in July 2010 to the present date, by quarter.

 

 

 

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