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Advances in cochlear gene therapies
Journal article   Open access   Peer reviewed

Advances in cochlear gene therapies

Miles J Klimara and Richard J H Smith
Current opinion in pediatrics, Vol.35(6), pp.631-640
12/2023
DOI: 10.1097/MOP.0000000000001273
PMCID: PMC10771539
PMID: 37417821
url
https://pmc.ncbi.nlm.nih.gov/articles/PMC10771539/pdf/nihms-1911964.pdfView
Open Access

Abstract

PURPOSE OF REVIEW Hearing loss is the most common sensory deficit and in young children sensorineural hearing loss is most frequently genetic in etiology. Hearing aids and cochlear implant do not restore normal hearing. There is significant research and commercial interest in directly addressing the root cause of hearing loss through gene therapies. This article provides an overview of major barriers to cochlear gene therapy and recent advances in preclinical development of precision treatments of genetic deafness. RECENT FINDINGS Several investigators have recently described successful gene therapies in many common forms of genetic hearing loss in animal models. Elegant strategies that do not target a specific pathogenic variant, such as mini gene replacement and mutation-agnostic RNA interference (RNAi) with engineered replacement, facilitate translation of these findings to development of human therapeutics. Clinical trials for human gene therapies are in active recruitment. SUMMARY Gene therapies for hearing loss are expected to enter clinical trials in the immediate future. To provide referral for appropriate trials and counseling regarding benefits of genetic hearing loss evaluation, specialists serving children with hearing loss such as pediatricians, geneticists, genetic counselors, and otolaryngologists should be acquainted with ongoing developments in precision therapies.

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