Novel AAV-based GJB2 gene therapy restores hearing function

Le résumé fourni par la source

Many scientists and clinicians consider GJB2 to be the “Holy Grail” of inner ear gene therapy. The gene encodes connexin 26, and its mutations are responsible for a large proportion of non-syndromic hearing loss. On a global scale, it represents one of the most prevalent reasons for prelingual severe to profound sensorineural hearing loss, leading to autosomal recessive deafness 1 (DFNB1). Because of how many patients could be treated and the potential impact of developing a therapy for such a significant sensory deficit, it has been one of the main targets pursued by researchers in academia and industry alike, but it has proven to be an elusive one.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.