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Experimental compound reverts condition similar to Down syndrome

09/05/2013

This article was translated by an automatic translation system, and was therefore not reviewed by people.

 

 

 



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Drugs administered in a single dose on the day of birth, normalized growth of the cerebellum and strengthened learning and memory in mice

Genetically modified mice tested new compound Photo : Robert Bukaty / AP
NEW YORK - Researchers from Johns Hopkins University and the National Institutes of Health have discovered a compound that enhances learning and memory when administered as a single dose on the day of birth in rodents with a condition like Down syndrome . The study was reported in Wednesday's edition of the journal " Science Translational Medicine" .

The treatment with the experimental compound appears to allow the cerebellum of rodents grow normally - the majority of people with Down syndrome is 60 % of the cerebellum - but still not a promise of a cure for syndrome, only one signal for the creation of new medications.

- We treat rodents with the condition like Down to normalize the growth of the cerebellum and it worked . Not expecting the developments in learning and memory , usually controlled by the hippocampus , the cerebellum does not - says Professor Roger Reeves , Institute of Genetic Medicine McKusick - Nathans , the School of Medicine at Johns Hopkins .

Reeves has devoted his career to studying Down syndrome , a condition that occurs when a person has three ( instead of two ) copies of chromosome 21 . As a result of trisomy , who has Down has extra copies of more than 300 genes housed in this chromosome , which leads to intellectual disabilities , spatial difficulties , facial features and , often , heart problems . With the involvement of many genes , develop a treatment is a major challenge , according to the researcher .

For this experiment Reeves and colleagues used genetically modified rodents with extra copy of about half of the genes found on chromosome 21 that mimics the characteristics of Down syndrome . Based on previous experiences on how Down syndrome affects brain development , the researchers tried to overload the biochemical chain of events that triggers growth and development . The compound was injected in rodents only once , on the day of birth, as her cerebellum were still developing . In addition to measuring the cerebellum , the team examined possible changes in behavior .

- Modify the animals , synthesize the compound and reach the right dose consumed was so hard and so long that we seek to achieve the maximum data - explains Reeves .

The team tested the modified rodents and compared with modified animals with Down but not treated , and mice without genetic modification . Rodents treated with the experimental compound fared so well in the test space , location in a water maze , as rodents without genetic modification to Down .
The researcher believes that further tests are needed to find out exactly why the treatment works , since examinations in some cells in the hippocampus , involved in learning showed no change with treatment. One hypothesis is that the treatment has improved learning by strengthening communication between the cerebellum and hippocampus.

As for the compound's potential to become a human medicinal product , the problem , Reeves says , is that the change of a biological chain of events would likely have important many unwanted effects throughout the body , such as increased risk of cancer , triggered by growth inappropriate .
And even though his team could develop a drug clinically useful , however , warns that Reeves did not constitute a "cure " for learning and memory-related effects of Down syndrome .


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