Genetic Therapies for Hearing Loss: Accomplishments and Remaining Challenges

Neurosci Lett. 2019 Nov 20;713:134527. doi: 10.1016/j.neulet.2019.134527. Epub 2019 Oct 3.


More than 15 years have passed since the official completion of the Human Genome Project. Predominantly due to this project, over one hundred genes have now been linked to hearing loss. Although major advancements have been made in the understanding of underlying pathologies in deafness as a consequence of these gene discoveries, biological treatments for these conditions are still not available and current treatments rely on amplification or prosthetics. A promising approach for developing treatments for genetic hearing loss is the most simplistic one, that of gene therapy. Gene therapy would intuitively be ideal for these conditions since it is directed at the very source of the problem. Recent achievements in this field in laboratory models spike hope and optimism among scientists, patients, and industry, and suggest that this approach can mature into clinical trials in the coming years. Here we review the existing literature and discuss the different aspects of developing gene therapy for genetic hearing loss.

Keywords: Cochlea; Deafness; Gene editing; Gene therapy; Hair cells; Inner ear.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Genetic Therapy / methods*
  • Genetic Vectors / therapeutic use
  • Hearing Loss / therapy*
  • Humans
  • Viruses / genetics