Work

Preclinical Models to Study the Molecular Pathophysiology of Meniere’s Disease: A Pathway to Gene Therapy

Year: 2025

Type: article

Abstract: Background: Meniere’s disease (MD) is a set of rare disorders that affects >4 million people worldwide. Individuals with MD suffer from episodes of vertigo associated with fluctuating sensorineural hearing loss and tinnitus. Hearing loss can involve one or both ears. Over 10% of the reported cases are observed in families, suggesting its significant genetic contribution. The condition is polygenic with >20 genes, and several patterns of inheritance have been reported, including autosomal dominant, autosomal recessive, and digenic inheritance across multiple MD families. Preclinical research using animal models has been an indispensable tool for studying the neurophysiology of the auditory and vestibular systems and to get a better understanding of the functional role of genes that are involved in the hearing and vestibular dysfunction. While mouse models are the most used preclinical model, this review analyzes alternative animal and non-animal models that can be used to study MD genes. Methods: A literature search of the 21 genes reported for familial MD and the preclinical models used to investigate their functional role was performed. Results: Comparing the homology of proteins encoded by these genes to other model organisms revealed Drosophila and zebrafish as cost-effective models to screen multiple genes and study the pathophysiology of MD. Conclusions: Murine models are preferred for a quantitative neurophysiological assessment of hearing and vestibular functions to develop drug or gene therapy.

Source: Journal of Clinical Medicine

DOI: 10.3390/jcm14051427

Authors: Prathamesh T. Nadar-Ponniah, Jose A. Lopez-Escamez

Institutions: The University of Sydney, Universidad de Granada, Centre for Biomedical Network Research on Rare Diseases, Instituto de Investigación Biosanitaria de Granada, Centro de Investigación Biomédica en Red

Language: English

Topic: Vestibular and auditory disorders

Subfield: Neurology

Field: Neuroscience

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,137