Article Dans Une Revue Molecular Therapy - Methods and Clinical Development Année : 2025

AAV-mediated inner ear gene delivery triggers mild host immune responses in the mammalian inner ear

Résumé

Hearing loss is a common disability affecting the world's population. Currently, its treatment options are limited. Adenoassociated virus (AAV)-mediated inner ear gene therapy has shown great promise as a treatment for hereditary hearing loss. However, the host immune responses to AAV-mediated gene therapy in the mammalian inner ear is not well understood. In this study, two serotypes of AAV vectors were injected individually into the mouse inner ear to evaluate the host innate and adaptive immune responses up to 1 month after inner ear gene delivery. Our results suggest that the host innate and adaptive immune responses to AAV-mediated inner ear gene delivery are limited and mild, which is favorable for its clinical translation.

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hal-05082187 , version 1 (23-05-2025)

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Yasuko Ishibashi, Jianliang Zhu, Gwladys Gernoux, Yunkai Yu, Michelle J Suh, et al.. AAV-mediated inner ear gene delivery triggers mild host immune responses in the mammalian inner ear. Molecular Therapy - Methods and Clinical Development, 2025, 33 (2), pp.101456. ⟨10.1016/j.omtm.2025.101456⟩. ⟨hal-05082187⟩
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