Gene Therapy for Children with Deafness: We Are Almost There, At Least for Single Gene Mutations

This year (2024), at various international audiology and otolaryngology conferences (e.g., AAA; ARO), we have heard about preliminary findings on the restoration of hearing in children after gene therapy. This follows from at least a decade of research studies in animal models of deafness due to single gene mutations where the correct gene insertion has been shown to be (a) possible, (b) safe, and (c) with some limited degree of efficacy. There are numerous up-to-date reviews of such studies.