Emerging Gene Therapies for Genetic Hearing Loss

J Assoc Res Otolaryngol. 2017 Oct;18(5):649-670. doi: 10.1007/s10162-017-0634-8. Epub 2017 Aug 16.


Gene therapy, or the treatment of human disease using genetic material, for inner ear dysfunction is coming of age. Recent progress in developing gene therapy treatments for genetic hearing loss has demonstrated tantalizing proof-of-principle in animal models. While successful translation of this progress into treatments for humans awaits, there is growing interest from patients, scientists, clinicians, and industry. Nonetheless, it is clear that a number of hurdles remain, and expectations for total restoration of auditory function should remain tempered until these challenges have been overcome. Here, we review progress, prospects, and challenges for gene therapy in the inner ear. We focus on technical aspects, including routes of gene delivery to the inner ear, choice of vectors, promoters, inner ear targets, therapeutic strategies, preliminary success stories, and points to consider for translating of these successes to the clinic.

Keywords: cochlea; gene therapy; genetic deafness; hair cell; inner ear; spiral ganglion neuron; vestibular; viral vectors.

Publication types

  • Review

MeSH terms

  • Animals
  • Genetic Therapy / methods*
  • Genetic Therapy / trends*
  • Genetic Vectors
  • Hearing Loss / genetics
  • Hearing Loss / therapy*
  • Humans
  • Translational Research, Biomedical