New research funding puts Rett Syndrome cure on the horizon | Business Standard

Researchers from Scotland and the US are taking a step towards reversing the effects of Rett Syndrome, a debilitating genetic disease affecting very young children in large numbers worldwide. 

Scientists from the University of Glasgow have been awarded USD 1.6 million to work together with three research institutes in the US to develop treatments for Rett Syndrome, a profoundly disabling condition that has commonalities with autism spectrum disorders. 

The grant, from the Rett Syndrome Research Trust (RSRT), will build an international consortium of researchers who will expand the breadth and depth of high-impact gene therapy research into the disease. 

This is the first research group in the world specialising in gene therapy to combat the disease. This consortium will build on recent research breakthroughs that have successfully used gene therapy to reverse the progress of the disease in mice. 

Future research will concentrate on developing similar treatments for clinical trials in humans. 

Rett Syndrome is caused by a mutation in the MECP2 gene that leads to numerous devastating symptoms, some of which worsen over time. 

It predominantly affects girls and causes severe regression in early childhood robbing children of the ability to speak, move normally and use their hands. 

The disease also causes disordered breathing, tremors, severe anxiety and emotional disturbances, seizures, and digestive, circulatory and orthopaedic problems. 

Although most children survive to adulthood they require total round-the-clock care for the duration of their lives. Researchers will target the underlying cause of the illness using gene therapy. 

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New research funding puts Rett Syndrome cure on the horizon | Business Standard