Can we develop enhanced treatments for fragile X syndrome?

Even in sleep, your brain’s neurons are humming along to Mozart
By Neuroscience Research Theme
1 min read

Fragile X syndrome is the leading inherited cause of intellectual disability and autism, but we still lack treatments that target its underlying biology. 

A new study from Carlos Portera-Cailliau's lab at David Geffen School of Medicine at UCLA (led by Anand Suresh) profiled gene activity in 2 key types of neurons in a mouse model and found that many signaling pathways were altered in opposite directions in excitatory versus inhibitory cells. 

This may explain the brain's excitation/inhibition imbalance. One gene stood out: Epac2. Reducing it genetically restored cortical balance and a drug that blocks EPAC2 improved circuit function and several behaviors in the mice. A promising new therapeutic target for fragile X. 

Read the entire paper: Translatome profiling reveals opposing alterations in inhibitory and excitatory neurons of fragile X mice and identifies EPAC2 as a therapeutic target