Practical aspects of inner ear gene delivery for research and clinical applications.

The application of gene therapy is widely expanding in research and continuously improving in preparation for clinical applications. The inner ear is an attractive target for gene therapy for treating environmental and genetic diseases in both the auditory and vestibular systems. With the lack of spontaneous cochlear hair cell replacement, hair cell regeneration in adult mammals is among the most important goals of gene therapy. In addition, correcting gene defects can open up a new era for treating inner ear diseases. The relative isolation and small size of the inner ear dictate local administration routes and carefully calculated small volumes of reagents. In the current review, we will cover effective timing, injection routes and types of vectors for successful gene delivery to specific target cells within the inner ear. Differences between research purposes and clinical applications are also discussed.
Competing Interests: Declaration of competing interest SG is on the advisory boards for the Cystic Fibrosis Foundation, Applied Genetic Technologies Corporation and Roche. He also receives research support without financial remuneration from Med-El Corporation and is a consultant for Cochlear Corporation and Sirocco Therapeutics. YR is on the advisory board of Applied Genetic Technologies Corporation.
(Copyright © 2020 Elsevier B.V. All rights reserved.)