Korean J Otorhinolaryngol-Head Neck Surg.  2021 Apr;64(4):215-222. 10.3342/kjorl-hns.2020.00836.

Inner Ear Gene Therapy in Mouse Models of Genetic Hearing Loss

  • 1Department of Otorhinolaryngology-Head and Neck Surgery, College of Medicine, Chungnam National University, Daejeon, Korea


Hearing loss is the most frequent sensory disorder affecting newborns and children. About 1 newborn in every 500 suffer from congenital hearing loss, with approximately half of these having a genetic cause. In the last few decades, the study of genetic hearing loss and related mouse models has unveiled molecular, cellular, and physiological mechanisms of the disease. In addition, effective and safe viral vectors for gene delivery to the inner ear have been generated. A growing number of approaches, including gene replacement, gene silencing, and gene editing, have proved effective in mouse models. This article briefly introduces basic strategies of gene therapy, viral vectors used and surgical methods for gene delivery, and reviews the current works on mouse modes of genetic hearing loss.


Animal model; Correction of hearing impairment; Gene therapy; Genetic vectors; Nonsyndromic sensorineural hearing loss; Sensorineural hearing loss
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