USHER-GT

Introduction

What is USHER-GT ?

Sensorion’s USHER-GT gene therapy development program aims to restore inner ear function for patients suffering from Usher Syndrome Type 1. By providing a healthy copy of the USH1G gene coding for the SANS protein (scaffold protein containing ankyrin repeats and SAM domain).

USH1G gene therapy restored hearing and vestibular functions

Proof of concept in a knock-out mouse model by Institut Pasteur

Source : Emptoz et al.,”Local Gene Therapy durably restores vestibular function in a mouse model of Usher syndrome type 1G” 2017 (link)

Mechanoelectrical transduction (MET) currents recorded ex vivo (recording of peak amplitude of the MET currents)

Source : Emptoz et al. 2017 (link)

Restoration of stereo cilia physiology using AAV8-SANS restored electrical excitability of sensory cells