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Molecular therapy for genetic and degenerative vestibular disorders

Sayyid, Zahra N.*; Kim, Grace S.*; Cheng, Alan G.

Current Opinion in Otolaryngology & Head and Neck Surgery: October 2018 - Volume 26 - Issue 5 - p 307–311
doi: 10.1097/MOO.0000000000000477

Purpose of review The primary purpose of this review is to summarize current literature in the field of vestibular regeneration with a focus on recent developments in molecular and gene therapies.

Recent findings Since the discovery of limited vestibular hair cell regeneration in mammals in the 1990s, many elegant studies have improved our knowledge of mechanisms of development and regeneration of the vestibular system. A better understanding of the developmental pathways of the vestibular organs has fueled various biological strategies to enhance regeneration, including novel techniques in deriving vestibular hair cells from embryonic and induced pluripotent stem cells. In addition, the identification of specific genetic mutations responsible for vestibular disorders has opened various opportunities for gene replacement therapy.

Summary Vestibular dysfunction is a significant clinical problem with limited therapeutic options, warranting research on biological strategies to repair/regenerate the vestibular organs to restore function. The use of gene therapy appears promising in animal models of vestibular dysfunction.

Department of Otolaryngology-Head and Neck Surgery, Stanford University School of Medicine, Stanford, California, USA

Correspondence to Alan G. Cheng, MD, Department of Otolaryngology-Head and Neck Surgery, 801 Welch Road, Stanford, CA 94305, USA. Tel: +1 (650) 725-6500; fax: +1 (650) 721-2163; e-mail:

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