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Drug Identified that May Help Prevent Down Syndrome

Down Syndrome, Trisomy 21

In a recent press release, the McKusick-Nathans Institute of Genetic Medicine at Johns Hopkins has received a grant in the amount of $250,000. This grant was provided by the Down Syndrome Research and Treatment Foundation, and will be used to further research into Down syndrome.

It is estimated that Down syndrome affects over 350,000 Americans. Physiology professor Roger H. Reeves, Ph.D., and a member of Johns Hopkins, will use this grant to further their studies into drug research for Down syndrome.

Using mice that are affected with Down syndrome, researchers are seeking a drug that will facilitate improvements in brain development.

The researchers had already discovered a particular drug that could contribute to making brain cells grow more normally. If the brain were to develop normally, this would decrease the probability of a child being born with Down syndrome.

They discovered that by injecting a drug named SAG, (or sonic agonist), one time causes the brain cells to have proper growth during it’s first third of development. Researchers plan to do additional studies using different amounts of the drug SAG.

Additionally, they intend to monitor brain cell development to see if this drug has any impact on any other part of the cells within the brain as they develop.

The mice that are being used in this study, and injected with the drug SAG, will also be tested to see whether or not there are any improvements in their memory skills or learning abilities than mice who are not given injections of SAG.

“The DSRTF award allows us to pursue our preliminary observations immediately without the now considerable delay that’s usually associated with obtaining federal funding. With a decrease of nearly 70 percent – from $42 million to $13 million – in the amount of Down syndrome research dollars from the National Institutes of Health in the last few years, the role of groups like DSRTF has become critical to continued progress in the effort to help 350,000 Americans with Down syndrome,” said Roger H. Reeves, Ph.D.

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When someone inherits three copies of chromosome 21, they develop a condition called trisomy 21 (or Ts21). This causes the part of the brain responsible for motor-learning and coordinating movement to grown very slowly, too slowly in most cases. The result? An under-developed and small brain.

During normal development, only two copies of chromosome 21 are inherited.
“DSRTF is extremely pleased to award this major new DSRTF research grant to Dr. Reeves, which will allow his research group to rapidly extend and expand their ground-breaking research to investigate a novel potential therapeutic target specifically related to an impairment in neurogenesis and brain development in Down syndrome,” said Dr. Michael Harpold, chief executive officer of the Down Syndrome Research and Treatment Foundation.

Source:
http://www.hopkinsmedicine.org/Press_releases/2007/10_31a_07.html