Down syndrome therapy now possible, says US researcher

Cross-posted from BioEdge

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.

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