Outside-in: Rethinking the etiology of autism spectrum disorders

Abstract

A role for peripheral sensory neurons in autism pathogenesisAutism spectrum disorders (ASDs) are thought to arise exclusively from aberrant brain function. Our research proposes a surprising revision of this view. We have discovered that peripheral sensory neurons—neurons outside the brain—are key sites at which ASD-related gene mutations have a critical impact. Dysfunction of peripheral neurons in mouse models disrupts central nervous system development and causes ASD-related phenotypes, including sensory overreactivity, social impairments, and anxiety-like behaviors. These unexpected findings have led us to propose and demonstrate that peripheral neurons are a tractable and effective therapeutic target to improve some ASD-related behaviors.