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Journal of the Association for Research in Otolaryngology

Erschienen in:

16.08.2017 | Review Article

Emerging Gene Therapies for Genetic Hearing Loss

verfasst von: Hena Ahmed, Olga Shubina-Oleinik, Jeffrey R. Holt

Erschienen in: Journal of the Association for Research in Otolaryngology | Ausgabe 5/2017

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Abstract

Gene therapy, or the treatment of human disease using genetic material, for inner ear dysfunction is coming of age. Recent progress in developing gene therapy treatments for genetic hearing loss has demonstrated tantalizing proof-of-principle in animal models. While successful translation of this progress into treatments for humans awaits, there is growing interest from patients, scientists, clinicians, and industry. Nonetheless, it is clear that a number of hurdles remain, and expectations for total restoration of auditory function should remain tempered until these challenges have been overcome. Here, we review progress, prospects, and challenges for gene therapy in the inner ear. We focus on technical aspects, including routes of gene delivery to the inner ear, choice of vectors, promoters, inner ear targets, therapeutic strategies, preliminary success stories, and points to consider for translating of these successes to the clinic.

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Titel: Emerging Gene Therapies for Genetic Hearing Loss
verfasst von: Hena Ahmed
Olga Shubina-Oleinik
Jeffrey R. Holt
Publikationsdatum: 16.08.2017
Verlag: Springer US
Erschienen in: Journal of the Association for Research in Otolaryngology / Ausgabe 5/2017
Print ISSN: 1525-3961
Elektronische ISSN: 1438-7573
DOI: https://doi.org/10.1007/s10162-017-0634-8

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Emerging Gene Therapies for Genetic Hearing Loss

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