More Déjà Vu...Autism Cured in Mice!

A group of scientists led by Susumu Tonegawa at MIT (Where have I heard that name before?) have demonstrated the first successful treatment for Fragile X Syndrome (FXS), a cause of mental retardation and autism. In mice genetically engineered as a model for FXS, they used genetic techniques to inhibit an enzyme called p21-activated kinase (PAK). Inhibiting PAK resulted in structural changes in the nerve cells as well as improvement of behavioral abnormalities. This is not to say that we will be able to cure autism by genetic engineering, but there are drugs that inhibit PAK, and they might be useful for treating some kinds of autism. Let me emphasize that FXS is only one cause of autism and mental retardation. The causes of the wide range of conditions along the autism spectrum remain unknown and have been the source of much controversy. An interesting dialogue about the subject, including whether autism should even be “cured” at all, can be found here. Here's the BBC story. Here's the original paper. Here's more than you would ever want to know about FXS.