Single Injection Reverses Down Syndrome in Newborn Mice


Fri, Sep 6th, 2013 10:00 by capnasty NEWS

While not ready yet for humans, scientists at the Johns Hopkins University School of Medicine were able to "reverse Down syndrome in newborn lab mice by injecting an experimental compound that causes the brain to grow normally."

The compound, which has not been proven safe for use in humans, is designed to boost normal growth of the brain and body via a gene known as SHH.

The gene provides instructions for making a protein called sonic hedgehog, which is essential for development.

"It worked beautifully," said Roger Reeves of the Johns Hopkins University School of Medicine.

"Most people with Down syndrome have a cerebellum that's about 60 per cent of the normal size," he said.

"We were able to completely normalise growth of the cerebellum through adulthood with that single injection."

The injection also led to unexpected benefits in learning and memory, normally handled by a different part of the brain known as the hippocampus.



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