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Compound normalizes brain structure, function in mice with Down syndrome, Kyoto researchers say

Sep 05, 2017 (Japan Times) - A team of Kyoto University researchers announced Tuesday that they have discovered a chemical compound that may help nerve cells grow in the brains of people with Down syndrome and improve their learning ability.

The findings could lead to the development of drugs to treat Down syndrome in fetuses, they said, adding that it could also lead to treatment for other cerebral nerve illnesses, including Alzheimer’s and Parkinson’s diseases.

Down syndrome, caused in most cases by an extra copy of chromosome 21, genetically impairs intellectual ability. At present, prenatal diagnosis is possible. But there are no therapies available now for normalizing brain functions.

Masatoshi Hagiwara, professor at Kyoto University, and colleagues identified the compound, called altered generation of neurons, or ALGERNON, after screening a total of 717 candidate compounds.

Re-creating cells of people with Down syndrome from induced pluripotent stem (iPS) cells, the researchers then confirmed that the compound inhibited the target gene from restricting proliferation of neural stem cells, which become nerve cells, and increased the number of newborn neurons to almost the level without the syndrome.

The oral administration of the compound to pregnant mice with the syndrome for five days also normalized the formation of the cerebral cortex in embryos and prevented the development of abnormal behavior in offspring, the researchers said.

Source: ANNnewsCH

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