A chromosome therapy for Down syndrome may be possible, according to ground-breaking research published in Nature. Lead author Jeanne B. Lawrence, of the University of Massachusetts Medical School, says that the "fix" silences the extra chromosome which causes DS.
"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 brings into the realm of consideration research on the concept of 'chromosome therapy' in the future," she says.
However, a cure is still a long way off, she says – at least ten years away. "I don't want to get people's hopes up."
But this is important research. "Since therapeutic strategies for common chromosomal abnormalities like Down syndrome have received too little attention for too long, for the sake of millions of patients and their families across the US and the world, we ought to try," Dr Lawrence says.
This article is published by Michael Cook
and BioEdge.org under a Creative Commons licence. You may republish it or translate it free of charge with attribution for non-commercial purposes following these guidelines
. If you teach at a university we ask that your department make a donation. Commercial media must contact us
for permission and fees. Some articles on this site are published under different terms.