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Gene therapy

Gene therapy for hereditary deafness

Bilateral administration of human OTOF gene therapy appears safe, and preliminary data suggest that it could effectively treat a rare form of hereditary hearing loss by enhancing speech perception, sound source localization and overall auditory experience.

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Fig. 1: Gene therapy via AAV injection into the inner ear.


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Correspondence to Mustafa Tekin.

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The authors declare no competing interests.

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Smith, C., Zafeer, M.F. & Tekin, M. Gene therapy for hereditary deafness. Nat Med (2024).

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