This is a novel strategy to treat tactile hypersensitivity, anxiety, and social interaction deficits by targeting the peripheral nervous system without drugging the brain.

Autism spectrum disorders (ASDs), including Rett Syndrome, as well as Fragile X, are characterized by abnormal social interactions and reactions to sensory stimuli. Research from the Ginty lab at Harvard Medical School, published on June 9, 2016 in Cell, suggests that the abnormal perception of touch and the presence of anxiety and social abnormalities, are linked to defects in specific peripheral nerve cells. Moreover, the mechanism mediating hypersensitivity, anxiety, and defects in social interaction was identified, and was found to be amenable to pharmaceutical intervention in the peripheral nervous system.

The mechanism and preliminary intervention were demonstrated in engineered mice that have, in their peripheral nervous system only, mutations known to be associated with ASD in humans, including MECP2, which causes Rett syndrome, GABRB3, implicated in ASD, and Fmr1, associated with Fragile X.

The Ginty lab is interested in a collaboration to use this new information about the biological mechanisms by which these syndromes are mediated to further develop therapeutic strategies for humans.