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

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...

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Shranjeno v:
Bibliografske podrobnosti
izdano v:Hum Mol Genet
Main Authors: Vijayakumar, Sarath, Depreux, Frederic F., Jodelka, Francine M., Lentz, Jennifer J., Rigo, Frank, Jones, Timothy A., Hastings, Michelle L.
Format: Artigo
Jezik:Inglês
Izdano: Oxford University Press 2017
Teme:
Articles
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC5886244/
https://ncbi.nlm.nih.gov/pubmed/28633508
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddx234
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