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McGill scientists link autistic behavior to brain protein mutation

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McGill University researchers and colleagues discovered a protein abnormality linked to symptoms of autism in mice. Interestingly, as Bloomberg reports, the scientists were able to use an experimental cancer drug to successfully reverse some of the autistic-like behaviors.

Details are published in the journal Nature.

As the story explains, the researchers started their work looking at genetically altered mice with major social problems (such as bad communication, social interaction and repetitive behaviors). The animals all had too many neuroligins, substances on the receiving end of a synapse that, in large numbers, can boost the number of synapses to the point of excess. They created an overproduction of neuroligins by putting the 4E-BP2 gene out of commission.

What is noteworthy is that the mutation might actually be reversible, according to the story. An experimental cancer drug helped reduce some of the excess synapses, after which the mice developed better social behavior over time. While the drug appeared too toxic for people, scientists argue that they could find other, related compounds that might work as an autism treatment. Similar drugs might already be available that work in people and address the same molecular pathway.

The bid to find treatments for autism remains controversial. It is also important to note that this treatment concept is early stage and years away from human testing. But the study, backed by the Canadian Institutes of Health Research and Autism Speaks, at least offers another possible course of action down the line.

- read the Bloomberg story
- here's the journal abstract

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