Efficiency of gene therapy for sensorineural hearing loss in mouse model: A meta-analysis

Objectives

Sensorineural hearing loss (SNHL) is a disorder characterized by the loss or impairment of cochlear hair cells or the auditory nerve. In recent years, gene therapy has emerged as a promising approach for SNHL treatment. The objective of this study is to evaluate the impact of gene therapy on the restoration or improvement of auditory function in mouse model with loss or impairment of hearing.

Methods

Studies with clear experimental designs, and auditory brainstem response (ABR) analysis as relevant outcome measures were included by searching PubMed, Scopus, and Web of Science databases. The PRISMA guideline was used for abstracting data and assessing data quality and validity. A quantitative synthesis was performed using a random effects model to examine the effect of gene therapy on auditory function in SNHL.

Results

Nine articles including 71 studies meeting the inclusion criteria were identified. These studies explored therapies targeting the TMC1, VGLUT3, USH1C, CLRN1, WHRN, and PJVK genes, with genetic material ranging from 1.8 × 10¹¹ and 1.4 × 10¹⁴ gc/mL being delivered to the inner ear through round window membrane, cochleostomy, or posterior semicircular canal injection methods. The hearing test results showed a significant mean difference of 26.91 dB (95% CI: 22.01–31.85) in favor of the experimental group.

Conclusions

Although promising results have been obtained regarding the potential success of gene therapy in SNHL, further investigation is needed to explore the long-term effects of gene therapy, treatment response rates, and the relationships between different genetic mutation types.