Scientists believe they are a step closer to "switching off" a chromosome that causes Down syndrome.
Researchers claim they have shown in principle that a natural "off switch"– usually used to decide the sex of a baby – could be used to neutralize the extra chromosome behind trisomy 21, better known as Down Syndrome.
The finding paves the way for researchers to study the cell pathologies and identify genome-wide pathways implicated in the disorder, which has up until now been elusive.
But writing in the journal Nature, they warn it is still only a "conceivable idea" that the gene could be switched off in humans.
Professor Jeanne Lawrence, of University of Massachusetts Medical School, who led the research, said she hoped it could lead to new therapeutic techniques.
"Our hope is that for individuals living with Down syndrome, this proof of principle opens up multiple exciting new avenues for studying the disorder now, and bringing into the realm the consideration research on the concept of 'chromosome therapy' in the future," Professor Lawrence said.
Her research uses a naturally occurring genetic switch, called the XIST gene, to turn off the entire chromosome in one go.
It showed that inserting the XIST gene into the extra copy of chromosome 21 had a similar switching-off effect in the stem cells taken from a Down sufferer.
Scientists said the technique could be used to treat other conditions Down sufferers face, such as diseases of the bone marrow.
Neil Brockdorff, Professor of Developmental Epigenetics at the University of Oxford, told The Times: "In the long term it may even be possible to make the correction in patient-specific cell lines, and then use these for cell therapy, treating specific problems that individuals experience."
The chief executive of the Down Syndrome Association said the findings have "serious implications" and could be of significant benefit.
"This is exciting new research," Carol Boys said.
"The findings could have serious implications for future work that may be of real benefit to people with Down syndrome."
This month a new non-invasive blood test for Down syndrome in unborn babies went on trial.
Prenatal patients at King’s College Hospital and Medway Maritime hospital in Chatham, Kent, are to be offered the £400 screening test, which researchers claim can identify with 99 per cent certainty whether their unborn child will have Down syndrome.
Children with Down syndrome are born with three copies of chromosome 21, rather than the usual two. There are 23 pairs in total.
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