Usher syndrome in the inner ear: Etiologies and advances in gene therapy

Evan M. de Joya, Brett M. Colbert, Pei Ciao Tang, Byron L. Lam, Jun Yang, Susan H. Blanton, Derek M. Dykxhoorn, Xuezhong Liu

Research output: Contribution to journalArticlepeer-review

Abstract

Hearing loss is the most common sensory disorder with ~466 million people worldwide affected, representing about 5% of the population. A substantial portion of hearing loss is genetic. Hearing loss can either be non-syndromic, if hearing loss is the only clinical manifestation, or syn-dromic, if the hearing loss is accompanied by a collage of other clinical manifestations. Usher syndrome is a syndromic form of genetic hearing loss that is accompanied by impaired vision associated with retinitis pigmentosa and, in many cases, vestibular dysfunction. It is the most common cause of deaf-blindness. Currently cochlear implantation or hearing aids are the only treatments for Usher-related hearing loss. However, gene therapy has shown promise in treating Usher-related retinitis pigmentosa. Here we review how the etiologies of Usher-related hearing loss make it a good candidate for gene therapy and discuss how various forms of gene therapy could be applied to Usher-related hearing loss.

Original languageEnglish (US)
Article number3910
JournalInternational journal of molecular sciences
Volume22
Issue number8
DOIs
StatePublished - Apr 2 2021

Keywords

  • Antisense oligonucleotides
  • CRISPR
  • Retinitis pigmentosa
  • Syndromic hearing loss

ASJC Scopus subject areas

  • Catalysis
  • Molecular Biology
  • Spectroscopy
  • Computer Science Applications
  • Physical and Theoretical Chemistry
  • Organic Chemistry
  • Inorganic Chemistry

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