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Recent development of AAV-based gene therapies for inner ear disorders

A Correction to this article was published on 08 July 2021

A Correction to this article was published on 10 August 2020

This article has been updated

Abstract

Gene therapy for auditory diseases is gradually maturing. Recent progress in gene therapy treatments for genetic and acquired hearing loss has demonstrated the feasibility in animal models. However, a number of hurdles, such as lack of safe viral vector with high efficiency and specificity, robust deafness large animal models, translating animal studies to clinic etc., still remain to be solved. It is necessary to overcome these challenges in order to effectively recover auditory function in human patients. Here, we review the progress made in our group, especially our efforts to make more effective and cell type-specific viral vectors for targeting cochlea cells.

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Fig. 1: Adeno-associated virus-inner ear-Atoh1 (AAV-ie-Atoh1) induces new hair cells (HCs) in vivo with stereocilia.
Fig. 2
Fig. 3: Different strategies are used in constructing the synthetic promoter.

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Acknowledgements

This work was supported by the National Natural Science Foundation of China, 81970878 and 31771130 (GZ), Shanghai Municipal Government, and ShanghaiTech University.

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Lan, Y., Tao, Y., Wang, Y. et al. Recent development of AAV-based gene therapies for inner ear disorders. Gene Ther 27, 329–337 (2020). https://doi.org/10.1038/s41434-020-0155-7

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