Combined AAV-mediated gene replacement therapy improves auditory function in a mouse model of human DFNB42 deafness.

ABSTRACT: Hearing loss is a common disorder affecting nearly 20% of the world's population. Recently, studies have shown that inner ear gene therapy can improve auditory function in several mouse models of hereditary hearing loss. In most of these studies, the underlying mutations affect only a small number of cell types of the inner ear (e.g., sensory hair cells). Here, we applied inner ear gene therapy to the Ildr1^{Gt(D178D03)Wrst} (Ildr1^w-/-) mouse, a model of human DFNB42, non-syndromic autosomal recessive hereditary hearing loss associated with ILDR1 variants. ILDR1 is an integral protein of the tricellular tight junction complex and is expressed by diverse inner ear cell types in the organ of Corti and the cochlear lateral wall. We simultaneously applied two synthetic adeno

SUBMITTER: Isgrig K 

PROVIDER: S-EPMC10492026 | biostudies-literature | 2023 Sep

REPOSITORIES: biostudies-literature