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Article

Rescue of peripheral vestibular function in Usher syndrome mice using a splice-switching antisense oligonucleotide.

Human molecular genetics - 15 Sept 2017

Vijayakumar Sarath, Depreux Frederic F, Jodelka Francine M, Lentz Jennifer J, Rigo Frank, Jones Timothy A, Hastings Michelle L

Abstract excerpt

Usher syndrome type 1C (USH1C/harmonin) is associated with profound retinal, auditory and vestibular dysfunction. We have previously reported on an antisense oligonucleotide (ASO-29) that dramatically improves auditory function and balance behavior in mice homozygous for the harmonin mutation Ush1c c.216G > A following a single systemic administration. The findings were suggestive of improved vestibular function;...

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Share to Cytoskeletal Proteins
Share to Evoked Potentials, Auditory

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Share to Oligonucleotides, Antisense

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Share to Retinal Degeneration

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Share to Vestibule, Labyrinth

Source

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Identifiers

PMID: 28633508
DOI: 10.1093/hmg/ddx234
PMCID: PMC5886244

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026. Manifest publication-graph-cohort-2026-09-05.1.

Source receipt: pubmed, retrieved 5 Sept 2026.

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