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Article

Antisense oligonucleotide therapy mitigates autosomal dominant progressive hearing loss in a murine model of human DFNA2.

Molecular therapy : the journal of the American Society of Gene Therapy - 3 Dec 2025

Jang Seung Hyun, Roh Jae Won, Oh Kyung Seok, Joo Sun Young, Kim Jung Ah, Kim Se Jin, Choi Jae Young, Jung Jinsei, Kim Yeonjoon, Bok Jinwoong, Gee Heon Yung

Abstract excerpt

Hearing loss is the most common sensory disorder, with a substantial proportion caused by genetic mutations. KCNQ4, a voltage-gated potassium channel highly expressed in cochlear outer hair cells, is a common genetic etiology implicated in autosomal dominant progressive hearing loss (DFNA2). The dominant-negative KCNQ4 p.W276S (c.827G>C) mutation represents a mutational hotspot in DFNA2, yet no effective...

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Share to Disease Models, Animal
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Genes, Dominant

Share to Hair Cells, Auditory, Outer

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Share to Hearing Loss, Sensorineural

Share to KCNQ Potassium Channels

Share to Oligonucleotides, Antisense

Source

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Identifiers

PMID: 40898620
DOI: 10.1016/j.ymthe.2025.08.044
PMCID: PMC12703183

Genes

KCNQ4

Variants

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026.

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