Experimental drugs aimed at correcting three rare conditions linked with autism are beginning early-stage human trials. These therapies are targeted at Rett syndrome, fragile X [1], and tuberous sclerosis complex. And the scientists involved in these programs say that they have data that suggests the treatments could reverse the damage wrought by the ailments, according to a report in MIT Technology Review.
Researchers at MIT say they were able to correct abnormal brain development [1] as well as faulty memory while reducing the number of seizures in mice engineered to develop fragile X by reducing the activity of a receptor called metabotropic glutamate receptor 5, or mGluR5, says the Review.
MIT neuroscientist Mark Bear has founded a company, Seaside Therapeutics, to test that approach in humans.
"The idea that you could reintroduce function is a sea-change event," said Emanuel DiCicco-Bloom, a scientist at University of Medicine and Dentistry of New Jersey, recently told colleagues.
- check out the article [2]in MIT Technology Review
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Reversal of Fragile X symptoms a possibility [3]
MIT scientists reverse autism, retardation in mice [4]