Inhibiting excess protein degradtion for fragile X syndrome

Inhibiting protein degradation via the ubiquitin proteasome system could help treat fragile X syndrome by normalizing excessive proteostasis.

In an FMR1-knockout mouse model of fragile X syndrome, hippocampal slice biophysical assays, RNA sequencing and proteomic analysis showed the hyperactive protein synthesis that is a hallmark of the disease is coupled to increased proteasomal activity that drives excessive degradation of synaptic proteins. ...