Researchers at MIT have been able to not only understand how Fragile X Syndrome manifests within the mouse brain structure, but also have found an inhibitor protein that actually reverses the physical abnormalities seen in Fragile X mice. The inhibitor actually works therapeutically, meaning it reverses/treats the abnormalities after they have already formed in the brain.
Fragile X Syndrome in humans is associated with symptoms ranging from mild learning disabilities to severe autism. Their hope is that this drug works in a similar manner in humans, providing a means of treating children and people with some types of learning disabilities. Imagine if autism became a mostly treatable state – where the brain is able to return to a normal structure through protein inhibition. This isn’t some kind of doping or reduction of functionality. This is blocking protein action to allow normal development.
If this works in humans in a similar manner this could be a major breakthrough. That’s a big if though – not everything in the mouse model works identically in the human model.