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

Hearing loss is a common disability affecting the world's population. Currently, its treatment options are limited. Adeno-associated 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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Publication Details

Journal
Molecular Therapy — Methods & Clinical Development
Published
2025-03-22
DOI
https://doi.org/10.1016/j.omtm.2025.101456
Citations
2
Primary Topic
Hearing, Cochlea, Tinnitus, Genetics
Type
article
Field-Weighted Citation Impact
2.93

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article

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

Kevin Isgrig, Thomas B. Friedman, Michelle J. Suh, Rafal T. Olszewski et al.
2 citations
Molecular Therapy — Methods & Clinical Development
Hearing, Cochlea, Tinnitus, Genetics
2.93
article

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

Kevin Isgrig, Thomas B. Friedman, Michelle J. Suh, Rafal T. Olszewski, Wade W. Chien, Yunkai Yu, Yasuko Ishibashi, Oumeya Adjali, M’hamed Grati, Gwladys Gernoux, Michael Hoa, Cao Liang, Jianliang Zhu
article en
2 citations

Abstract

Hearing loss is a common disability affecting the world's population. Currently, its treatment options are limited. Adeno-associated 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.

Molecular Therapy — Methods & Clinical DevelopmentVol. 33(2)
National Institutes of Health (US), Johns Hopkins University (US), Inserm (FR), Johns Hopkins Medicine (US), Office of the Director (US), National Cancer Institute (US), National Institute on Deafness and Other Communication Disorders (US), Nantes Université (FR)
National Institute on Deafness and Other Communication Disorders
Openalex Percentile: Top 12%
Hearing, Cochlea, Tinnitus, Genetics
2.93
DOIOpen Access PDFOpenAlex
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