Drug Cures Mice Of Down Syndrome With A Single Dose 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. After a single injection of SAG on the day the mice were born their cerebellums developed normally into adulthood. It improved their behavior too: Mice treated with SAG performed just as well as normal mice on a memory and learning test. But the drug is a long way from becoming a human cure. The Hedgehog pathway plays an important role when it comes to brain development so fiddling with it could have unintended consequences. It's possible enhancing the biochemical events that lead to growth in the brain would cause issues elsewhere in the body like potentially raising the risk of cancer. Down syndrome is very complex and nobody thinks there's going to be a silver bullet that normalizes cognition Reeves said in a statement. Multiple approaches will be needed.The study appears in ABC.net.au]Ok.  I have two questions.  Don't hate me.  Inquiring minds really want to know.  What about the physical characteristics of Downs syndrome?  Will the child still have mongoloid characteristics but just be smarter?question 2.  Is that mouse's name Algernon?Q1) No
Q2) when we asked the mouse she simply stared. (jk and nice reference)Q!)sorry. Down Syndrome creates a great many genetic expressions to the individual. some dont get all that are possible. most likely any physiological characteristics would be largely permanent after birth. However if a drug were given in utero it potentially could make a difference as it might alter the genes before they were expressed. Maybe.
The treatment could also create any number of problems being given in utero. this injection was given after the mice were born and the article states that they were bred with similar characteristic to Down's not actually having Down's.
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