Gene therapy plus an injection of copper dramatically improved survival in mice with a condition that mimics the often fatal childhood disorder Menkes disease, according to a study by researchers at the National Institutes of Health. Menkes disease results from a poorly functioning copy of the gene ATP7A. Ordinarily, the gene helps the body process the trace metal copper. In Menkes disease, the gene malfunctions. As a result, copper is not available for proper development. Although the disorder affects the entire body, it is most pronounced in the brain, which requires relatively larger amounts of copper during development than other organs and tissues. Copper is used in the formation of red blood cells, and for keeping nervous system tissue, bones, and the immune system healthy.
