Emerging Therapies for Genetic Deafness: The Rise of Precision Gene Editing
Abstract
Hearing loss is a prevalent sensory disorder with profound social and health impacts. Although hearing aids and cochlear implants can significantly restore auditory perception, they do not address the underlying genetic causes. Inherited forms of hearing loss, often monogenic, are ideal candidates for gene therapy, particularly since over 150 genes have been linked to deafness. Advances in vector engineering and genome editing technologies now enable precise targeting of auditory cells within the inner ear. Technologies such as Clustered Regularly Interspaced Short Palindromic Repeats-Cas systems, base editing, and prime editing offer powerful tools to correct pathogenic variants at their native genomic loci. This review summarizes the current landscape of gene editing approaches for both hereditary and acquired hearing loss, emphasizing key preclinical studies in mouse models encompassing dominant and recessive forms of deafness. We discuss the advantages and limitations of different editing techniques, vector delivery systems, and administration routes, and explore obstacles including editing efficiency, cell-specific targeting, and the translation of these therapies to humans. Collectively, these advances represent a pivotal step toward variant-specific, durable, and potentially curative treatments for hearing loss.
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BibTeX
@article{lahlou2026emerging,
title = {{Emerging Therapies for Genetic Deafness: The Rise of Precision Gene Editing}},
author = {Ghizlene Lahlou and Maxence Cornille and Nicolas Lai and Marie-José Lecomte and Saaid Safieddine},
journal = {Ear \& Hearing},
year = {2026},
month = oct,
doi = {10.1097/aud.0000000000001903},
url = {https://hal.science/hal-05805399v1},
note = {CC BY-NC-ND 4.0},
}
RIS
TY - JOUR
TI - Emerging Therapies for Genetic Deafness: The Rise of Precision Gene Editing
AU - Ghizlene Lahlou
AU - Maxence Cornille
AU - Nicolas Lai
AU - Marie-José Lecomte
AU - Saaid Safieddine
PY - 2026
DA - 2026/10/13
JO - Ear & Hearing
DO - 10.1097/aud.0000000000001903
UR - https://hal.science/hal-05805399v1
AB - Hearing loss is a prevalent sensory disorder with profound social and health impacts. Although hearing aids and cochlear implants can significantly restore auditory perception, they do not address the underlying genetic causes. Inherited forms of hearing loss, often monogenic, are ideal candidates for gene therapy, particularly since over 150 genes have been linked to deafness. Advances in vector engineering and genome editing technologies now enable precise targeting of auditory cells within the inner ear. Technologies such as Clustered Regularly Interspaced Short Palindromic Repeats-Cas systems, base editing, and prime editing offer powerful tools to correct pathogenic variants at their native genomic loci. This review summarizes the current landscape of gene editing approaches for both hereditary and acquired hearing loss, emphasizing key preclinical studies in mouse models encompassing dominant and recessive forms of deafness. We discuss the advantages and limitations of different editing techniques, vector delivery systems, and administration routes, and explore obstacles including editing efficiency, cell-specific targeting, and the translation of these therapies to humans. Collectively, these advances represent a pivotal step toward variant-specific, durable, and potentially curative treatments for hearing loss.
SN - 1538-4667
SN - 0196-0202
ER -