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), [MORE DESCRIPTION ON THERAPEUTIC APPROACH].

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