Gene Therapy for Non-Syndromic Hearing Loss
DOI:
https://doi.org/10.71321/703b4949Keywords:
non-syndromic hearing loss, gene therapy, OTOF, DFNB9Abstract
Hereditary hearing loss accounts for over 60% of congenital deafness cases, with non-syndromic hearing loss (NSHL) representing the most common subtype. Typically caused by monogenic mutations, NSHL presents a promising candidate for gene therapy. Recent advances in deciphering the genetic underpinnings of deafness and developing gene delivery systems have greatly accelerated the progress of inner ear gene therapy, leading to a number of breakthrough achievements. This review provides an overview of the latest developments in gene therapy for NSHL. After outlining the genetic basis of NSHL, we summarize the preclinical progress made during the first decade of hereditary deafness gene therapy. Special emphasis is placed on gene replacement strategies for DFNB9, an autosomal recessive form of hearing loss caused by mutations in the OTOF gene. We highlight the remarkable journey of OTOF gene therapy and discuss future directions in this transformative field.
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