[Gene therapy progress: hopes for Usher syndrome]

Abstract

Hearing and balance impairment are major concerns and a serious public health burden, as it affects millions of people worldwide, but still lacks an effective curative therapy. Recent breakthroughs in preclinical and clinical studies using viral gene therapy suggest that such an approach might succeed in curing many genetic diseases. Our actual understanding and the comprehensive analysis of the molecular bases of genetic deafness forms have provided the multiple bridges toward gene therapy to correct, replace, or modify the expression of defective endogenous genes involved in deafness. The aim of this review article is to summarize the recent advances in the restoration of cochlear and vestibular functions by local gene therapy in mouse models of Usher syndrome, the leading genetic cause of deafness associated with blindness in the world. We focus herein on therapeutic approaches with the highest potential for clinical application.