Treatment following Triple-AAV Delivery in Mature Murine Model of Human CDH23-Associated Hearing Loss.

ABSTRACT: This study aimed to investigate the transduction efficiency of triple adeno-associated virus (AAV) vectors in the cochleae of adult mice, focusing on large-gene-associated hearing loss (HL). Additionally, we sought to evaluate the feasibility of cochlear gene therapy in a mouse model of human CDH23-mediated HL using the triple AAV approach. To create a reporter protein, we fused EGFP to mCherry, which was then divided into three parts, each packaged in a separate AAV2/2 vector. Four weeks after co-injecting the triple AAV vectors into 4-5-week-old mice, we assessed transduction efficiency. We found that up to 5.9% of inner hair cells were positive for both EGFP and mCherry. Subsequently, we developed triple Cdh23 AAV vectors for therapeutic purposes. After administering these

SUBMITTER: Yoshimura H 

PROVIDER: S-EPMC10741579 | biostudies-literature | 2023 Nov

REPOSITORIES: biostudies-literature