Scientists from Glasgow University have been awarded almost £1 million to work with three research institutes in the US to develop treatments for Rett Syndrome, which affects people at an early age and robs them of the ability to speak and move normally.
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 condition.
This consortium will build on research breakthroughs that have used gene therapy to reverse the progression of the disease in mice. Future research will concentrate on developing similar treatments for trials in humans.
Rett Syndrome is caused by a mutation in the MECP2 gene. It predominantly affects girls and causes severe regression in toddlerhood along with disordered breathing, tremors, anxiety and emotional disturbances, seizures and other problems. Although most children survive to adulthood, they require round-the-clock care for the rest of their lives.
Dr Stuart Cobb, a neuroscientist leading the team, said: "Gene therapy now gives us an arsenal of new weapons to combat this disease and it is conceivable that one day we will be able to correct the cause of Rett at its very source."