Advancing precision ear medicine: leveraging animal models for disease insights and therapeutic innovations.

ABSTRACT: Gene therapy offers significant promise for treating inner ear disorders, but its clinical translation requires robust preclinical validation, often reliant on animal models. This review examines the role of these models in advancing gene therapeutics for inherited inner ear disorders, focusing on successes, challenges, and treatment solutions. By providing a precise understanding of disease mechanisms, these models offer a versatile preclinical platform that is essential for assessing and validating therapies. Successful gene supplementation and editing have shown potential in restoring hearing and balance functions and preventing their decline. However, challenges such as limitations in gene delivery methods, surgical access, immune responses, and discrepancies in disease manifestation b

SUBMITTER: Vitry S 

PROVIDER: S-EPMC12130096 | biostudies-literature | 2025 Jun

REPOSITORIES: biostudies-literature