Down syndrome cells suppressed by scientists

U.S. researchers have discovered a way to disable the gene that causes Down syndrome, opening up new avenues for research and treatments.

In a study published Wednesday in Nature, scientists at the University of Massachusetts Medical Schoolsay they've found a way to suppress the effects of the extra chromosomethat causes a genetic disorder affecting 500 Canadian newborns each year.

"Our hope is that for individuals living with Down syndrome, this proof-of-principal 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," saidJeanne Lawrence, the lead author of the study.

Humans are typically born with46 chromosomes in each cell, but Down syndrome occurs whenan extra copy of chromosome21 is produced. This "trisomy 21" can lead to cognitive disabilities, early-onset Alzheimer's disease, infertility, shortened life expectancy and many other health effects.

Using lab-grown stem cells with Down syndrome, Lawrence's team spliced in an RNAgene called Xist, which normally functions to muzzle one of the two X chromosomes found in female embryos.

Xist functioned tomodify thestructure of the extra chromosome to deactivate most of its genes and prevent the production of proteins and other elements.

"Its a strategy that can be applied in multiple ways, and I think can be useful right now," Lawrence said.