Cure Down syndrome with a single injection? Well, maybe–if you’re a mouse. A team of scientists from John Hopkins University and the National Institutes of Health have cured newborn mice of Down syndrome by injecting them with a drug that stimulates what’s called the Sonic Hedgehog pathway (so-named because in flies, a lack of the Hedgehog signaling protein causes embryos to become prickly, hedgehog-like balls).
People with Down syndrome usually have smaller brain volumes than control groups, including significantly smaller cerebellums, a portion of the brain involved in motor control. The researchers, led by Roger Reeves of the John Hopkins University School of Medicine, treated newborn mice that had been genetically engineered to have Down syndrome-like characteristics with a small molecule called SAG.
Excerpt from an article written by Shaunacy Ferro at POPSCI. Continue THERE