Portraying inhibition of metabotropic glutamate receptor 5 in fragile X mice

摘要

The human genome project and genetic mapping of various common disorders have been considered to have opened a new area in our understanding of the pathophysiology of human illnesses and their treatment. However, the polygenic nature of the vast majority of diseases, interactions between multiple genes and genetic modifiers, as well as epigenetic factors represent a high level of complexity that has turned out to be a real barrier for developing effective therapies on the basis of genetic observations. Monogenic disorders, such as fragile X syndrome (FXS), represent the most promising target for potential drug treatments, given the relatively straightforward cellular/ biochemical consequences of the aberrant gene. The article in this issue of Biological Psychiatry by Michalon ef al. (1) is a direct result of the genetic studies of FXS and subsequent understanding of the physiological role of fragile X mental retardation protein (FMRP), eventually providing a "druggable" target to alleviate this devastating disorder.