New therapies possible for Down syndrome.

• Position: YOUR LIFE - Brief article

Conventional wisdom among scientists for years has suggested that, because individuals with Down syndrome have an extra chromosome, the disorder most likely results from the presence of too many genes or proteins contained in that additional structure. However, a study at Ohio State University, Columbus, reveals that just the opposite could be true--that a deficiency of a protein in the brain of Down syndrome patients may contribute to the cognitive impairment and congenital heart defects that characterize the condition.

Researchers have shown that manually manipulating pieces of RNA that regulate the protein could increase protein levels in the lining of brain cells. When this RNA segment is overexpressed--meaning that more of it is present than needed in a cell--the protein level goes down, or is underexpressed. A total of at least five of these RNA segments are overexpressed in persons with Down syndrome because the segments are...