Scientists Discover Potential Path to Treating Down Syndrome

Scientists are laying the groundwork for treating one of the most common genetic conditions in humans. Research out today highlights a promising new approach to Down syndrome, also known as trisomy 21. Neurologists at Beth Israel Deaconess Medical Center and Harvard Medical School have developed a new way to possibly repair the genetic error responsible for causing Down syndrome, a third copy of the 21st chromosome. Using a modified version of the gene-editing technique CRISPR/Cas9, they were able to silence this extra copy in a substantial number of cells in the lab. Though only a proof of concept for now, the researchers are hopeful their work can eventually lead to the first genuine treatment for the genetic disorder. “This approach overcomes a major hurdle,” senior study author Volney Sheen told Gizmodo. Down syndrome is estimated to occur in one of every 700 births in the U.S. The condition typically happens by random chance, though factors like older maternal age can increase its risk (the extra copy usually originates from the mother’s side). The most common form, accounting for 95% of cases, causes the third copy of the 21st chromosome to appear in all cells; this form is c