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유전성 난청 마우스 모델에서의 내이 유전자 치료

Inner Ear Gene Therapy in Mouse Models of Genetic Hearing Loss

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초록

Hearing loss is the most frequent sensory disorder affecting newborns and children. About 1newborn in every 500 suffer from congenital hearing loss, with approximately half of thesehaving a genetic cause. In the last few decades, the study of genetic hearing loss and relatedmouse 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 geneediting, have proved effective in mouse models. This article briefly introduces basic strategiesof gene therapy, viral vectors used and surgical methods for gene delivery, and reviews thecurrent works on mouse modes of genetic hearing loss.

Abstract

Hearing loss is the most frequent sensory disorder affecting newborns and children. About 1newborn in every 500 suffer from congenital hearing loss, with approximately half of thesehaving a genetic cause. In the last few decades, the study of genetic hearing loss and relatedmouse 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 geneediting, have proved effective in mouse models. This article briefly introduces basic strategiesof gene therapy, viral vectors used and surgical methods for gene delivery, and reviews thecurrent works on mouse modes of genetic hearing loss.

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