Ear and hearingOct 13, 2026

New Gene Editing Treatments for Inherited Deafness

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗OA ↗

Updated Oct 14, 2026

Abstract

Over 150 genes have been linked to deafness, highlighting the potential for gene therapy in treating inherited hearing loss.

Inherited forms of hearing loss are often caused by single gene mutations, making them suitable for gene therapy.
Recent advancements in gene editing technologies allow for precise corrections of genetic variants in auditory cells.
Techniques such as CRISPR-Cas systems, base editing, and prime editing can target specific genes within the inner ear.
Preclinical studies in mouse models show promise for addressing both dominant and recessive types of deafness.
Challenges remain in editing efficiency, targeted delivery to specific cells, and translating therapies for human use.

Simplified

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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New Gene Editing Treatments for Inherited Deafness

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