Mice with a Down’s-like Syndrome Respond Well to SHP-Agonist Treatment
A research collaboration between the John Hopkins Institute and the National Institutes of Health has shown that a single-dose injection given to mice at birth can reverse some of the symptoms of a condition similar to Down’s syndrome. The injection was found to boost learning and memory in the mice as well as leading to their cerebellums (the part of the brain responsible for coordination and motor control) maturing to a normal size in adulthood.
The study, reported in the latest issue of Science Translational Medicine, involved a small-molecule compound called a sonic hedgehog pathway agonist. People who suffer with Down’s syndrome have a cerebellum that is 40% smaller than a non-sufferer. The treatment not only led to the cerebellum maturing to full-size, it also led to enhancements in the function of the hippocampus which improved the learning and memory abilities of the mice.
Down’s syndrome is caused by three copies of chromosome 21 being present within an individual, instead of the usual two. This is known as trisomy and it means that in the case of Down’s syndrome there are extra copies of over 300 genes present which leads to the intellectual impairment, distinctive facial characteristics and health problems characteristic of Down’s. Treatment is hard as the symptoms are so wide-ranging.
The study team are keen to stress that this treatment is not remotely near to being a “magic cure” for Down’s syndrome; something which researchers believe is just not possible anyway. It should also be noted that although the mice had a condition similar to Down’s syndrome, they only had a 50% trisomy. The main problem with putting this compound into humans is that it alters a very specific chain of events in the development of the brain, which could lead to inappropriate growth and the development of cancers. The treatment does show, however, that treatment for the syndrome may be possible as long as the treatment is heavily specific and targeted to the right areas of the brain.
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