One Dose Of This Drug Cures Down Syndrome.. In Mice
Sep 10, 2013 10:53
Modern medicine is making more impactful headlines than Miley Cyrus this week. First it was a vaccine to prevent HIV/AIDS in the works, then tiny living 3d printed kidneys, and now, a new drug to cure Down Syndrome.
Scientists at the John Hopkins University and the National Institutes of Health have used a new drug to cure Down Syndrome in baby mice with just one dose. The drug has not been tested on humans just yet, but represents a huge breakthrough.
People born with down syndrome have extra copies of chromosomes. They cause problems with physical growth, along with severe learning disabilities. The part of the brain responsible for motor control is generally smaller than normal people.
Mice were engineered with Down Syndrome like characteristics and then injected with a drug shortly after their birth. It consists of a molecule called SAG that activates a particular pathway in the brain and stimulates the brain to grow to a normal size. In one dose, the brains of the mice grew normally and they showed learning abilities of its un-affected counterparts.
The SAG injection only worked on mice when used right after birth, and since mice aren't people, this injection doesn't deal with the extra chromosomes humans with Down Syndrome are born with.
Still, this is a huge achievement, and with any luck, we'll be one step closer to eradicating it.
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