Advancing precision ear medicine: leveraging animal models for disease insights and therapeutic innovations DOI Creative Commons
Sandrine Vitry, Clara Mendia, Audrey Maudoux

et al.

Mammalian Genome, Journal Year: 2025, Volume and Issue: unknown

Published: April 22, 2025

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 inherited focusing successes, challenges, and treatment solutions. By providing a precise understanding disease mechanisms, offer versatile platform that is essential assessing validating therapies. Successful supplementation editing have shown potential restoring hearing balance functions preventing their decline. However, challenges such as limitations delivery methods, surgical access, immune responses, discrepancies manifestation between humans hinder translation. Current efforts are dedicated to developing innovative strategies aimed at enhancing efficiency delivery, overcoming physical barriers blood-labyrinth barrier, improving target specificity, maximizing therapeutic efficacy while minimizing adverse responses. Diverse strategies, along with evolving technologies, hold outcomes using relevant The future will hinge personalized therapies team science fueling interdisciplinary collaborations among researchers, clinicians, companies, regulatory agencies expedite from bench bedside unlock immense precision medicine ear.

Language: Английский

Advancing precision ear medicine: leveraging animal models for disease insights and therapeutic innovations DOI Creative Commons
Sandrine Vitry, Clara Mendia, Audrey Maudoux

et al.

Mammalian Genome, Journal Year: 2025, Volume and Issue: unknown

Published: April 22, 2025

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 inherited focusing successes, challenges, and treatment solutions. By providing a precise understanding disease mechanisms, offer versatile platform that is essential assessing validating therapies. Successful supplementation editing have shown potential restoring hearing balance functions preventing their decline. However, challenges such as limitations delivery methods, surgical access, immune responses, discrepancies manifestation between humans hinder translation. Current efforts are dedicated to developing innovative strategies aimed at enhancing efficiency delivery, overcoming physical barriers blood-labyrinth barrier, improving target specificity, maximizing therapeutic efficacy while minimizing adverse responses. Diverse strategies, along with evolving technologies, hold outcomes using relevant The future will hinge personalized therapies team science fueling interdisciplinary collaborations among researchers, clinicians, companies, regulatory agencies expedite from bench bedside unlock immense precision medicine ear.

Language: Английский

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