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Aurora magazine

A new drug against autism is being tested

A study led by Professor Stuart Lipton is testing the effectiveness of a new drug against autism, NitroSynapsin. For the time being, the team is running tests on animal models, but the first results seem to be promising. The drug corrected a good part of the brain and behavioral abnormalities of the mice, with positive consequences on the typical symptoms of the autistic spectrum.

The new study is rooted in a 1993 study. Lipton and his Harvard team identified a gene involved in brain development, MEF2C. They later discovered that guinea pigs with an abnormal version of MEF2C showed symptoms similar to those of autism. This led to the identification of a similar genetic abnormality in human children affected by some forms of autism.

The MEF2C gene encodes a protein that acts as a transcription factor and is more or less directly involved in many forms of autism. Restoring gene functions should therefore have a positive effect on the disease. The drug NitroSynapsin serves to balance inhibitory signaling factors and an exciting inside the brain, abnormal in those suffering from some forms of autism.

The researchers gave the drug to guinea pigs for three months. Mice responded well to treatment. In many of them he reduced abnormal behaviors and improved cognitive and behavioral functions. In some cases, the guinea pigs are almost back to normal levels. The next step is to check on what forms of autism NitroSynapsin is effective what the effects on the human being are.