Immune responses in the mammalian inner ear and their implications for AAV-mediated inner ear gene therapy.

Autor: Ishibashi Y; Inner Ear Gene Therapy Program, National Institute on Deafness and Other Communication Disorders (NIDCD), National Institutes of Health, 35A 1F220, 35A Covent Dr., Bethesda, MD 20892, USA; Laboratory of Molecular Genetics, National Institute on Deafness and Other Communication Disorders (NIDCD), National Institutes of Health, Bethesda, MD, USA., Sung CYW; Laboratory of Hearing Biology and Therapeutics, National Institute on Deafness and Other Communication Disorders (NIDCD), National Institutes of Health, Bethesda, MD, USA., Grati M; Inner Ear Gene Therapy Program, National Institute on Deafness and Other Communication Disorders (NIDCD), National Institutes of Health, 35A 1F220, 35A Covent Dr., Bethesda, MD 20892, USA., Chien W; Inner Ear Gene Therapy Program, National Institute on Deafness and Other Communication Disorders (NIDCD), National Institutes of Health, 35A 1F220, 35A Covent Dr., Bethesda, MD 20892, USA; Department of Otolaryngology-Head & Neck Surgery, Johns Hopkins School of Medicine, Baltimore, MD, USA. Electronic address: wade.chien@nih.gov.
Jazyk: angličtina
Zdroj: Hearing research [Hear Res] 2023 May; Vol. 432, pp. 108735. Date of Electronic Publication: 2023 Mar 11.
DOI: 10.1016/j.heares.2023.108735
Abstrakt: Adeno-associated virus (AAV)-mediated inner ear gene therapy is a promising treatment option for hearing loss and dizziness. Several studies have shown that AAV-mediated inner ear gene therapy can be applied to various mouse models of hereditary hearing loss to improve their auditory function. Despite the increase in AAV-based animal and clinical studies aiming to rescue auditory and vestibular functions, little is currently known about the host immune responses to AAV in the mammalian inner ear. It has been reported that the host immune response plays an important role in the safety and efficacy of viral-mediated gene therapy. Therefore, in order for AAV-mediated gene therapy to be successfully and safely translated into patients with hearing loss and dizziness, a better understanding of the host immune responses to AAV in the inner ear is critical. In this review, we summarize the current knowledge on host immune responses to AAV-mediated gene therapy in the mammalian inner ear and other organ systems. We also outline the areas of research that are critical for ensuring the safety and efficacy of AAV-mediated inner ear gene therapy in future clinical and translational studies.
Competing Interests: Declaration of Competing Interest The authors have no conflict of interest to declare.
(Copyright © 2023. Published by Elsevier B.V.)
Databáze: MEDLINE
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