Rescue mission

A pair of preclinical studies published last month on the autism target SHANK3 provide what might be the most meaningful progress for patients since the gene was linked with the disease over a decade ago. While one paper showed symptoms could be reversed even in adult animals, the other provided the first small molecule strategy for preventing the synaptic dysfunction widely considered to lie at the root of the pathology.

Although SHANK3 has been well characterized as a synaptic protein, and is associated genetically with about 1% of all autism spectrum disorder patients, the target has mainly been exploited for creating animal models of disease, in part because its pathway has been difficult to attack therapeutically. ...