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).
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)
Source : Emptoz et al. 2017 (link)