Down’s syndrome cells ‘fixed’ in first step towards chromosome therapy

Down’s syndrome cells ‘fixed’ in first step towards chromosome therapy.

Researchers shut down the extra chromosome responsible for Down’s syndrome, paving the way for future treatments

Scientists have corrected the genetic fault that causes Down’s syndrome – albeit in isolated cells – raising the prospect of a radical therapy for the disorder.

In an elegant series of experiments, US researchers took cells from people with DS and silenced the extra chromosome that causes the condition. A treatment based on the work remains a distant hope, but scientists in the field said the feat was the first major step towards a “chromosome therapy” for Down’s syndrome.

“This is a real technical breakthrough. It opens up whole new avenues of research,” said Elizabeth Fisher, professor of neurogenetics at UCL, who was not involved in the study. “This is really the first sniff we’ve had of anything to do with gene therapy for Down’s syndrome.”

Around 750 babies are born with DS in Britain each year while globally between one in a 1000 and one in 1100 births are DS babies. Most experience learning difficulties.

via Down’s syndrome cells ‘fixed’ in first step towards chromosome therapy.

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