AAV-mediated inner ear gene delivery triggers mild host immune responses in the mammalian inner ear
Year: 2025
Type: article
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.
Institutions: National Institutes of Health, National Institute on Deafness and Other Communication Disorders, Inserm, Centre Hospitalier Universitaire de Nantes, Translational Research in Gene Therapy, Nantes Université, National Cancer Institute, Center for Cancer Research, Johns Hopkins University, Johns Hopkins Medicine
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $3,900
Awards: G334765041, G4595973020
OpenAlex ID: W4408738520