REVIEW article
Front. Cell Dev. Biol.
Sec. Stem Cell Research
Volume 13 - 2025 | doi: 10.3389/fcell.2025.1547523
This article is part of the Research TopicStudying Rare Diseases Using induced Pluripotent Stem Cell (iPSC)-based Model SystemsView all 5 articles
Current approaches for Usher syndrome disease models and developing therapies
Provisionally accepted
Fiona Kathryn Leith1,2
Joey Lye1,2
Derek Songjaroen Delaney1,2,3
Samuel Mclenachan2,4
Fred K. Chen2,4,5,6
Marcus D Atlas1,2
Elaine Y. Wong1,2,3*- 1Ear Science Institute Australia, Subiaco, Australia
- 2University of Western Australia, Perth, Western Australia, Australia
- 3Curtin University, Perth, Western Australia, Australia
- 4Lions Eye Institute, Perth, Western Australia, Australia
- 5Royal Perth Hospital, Perth, Western Australia, Australia
- 6The University of Melbourne, Parkville, Victoria, Australia
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Usher syndrome is a severely debilitating autosomal recessive disorder characterized by congenital or progressive hearing loss, gradual vision loss and in some subtypes, vestibular dysfunction. Much progress has been made in recent years in creating appropriate preclinical models for most subtypes of Usher syndrome to facilitate the development of novel therapies. In this review, we provide an update on new preclinical models of Usher syndrome, with a particular focus on induced pluripotent stem cells and new organoid models. An update on the status of novel therapies is provided, including the development of new genetic therapies using new preclinical models and those currently in clinical trials.
Keywords: Usher syndrome, Hearing Loss, Inner ear, Hair cell, Gene Therapy
Received: 18 Dec 2024; Accepted: 29 May 2025.
Copyright: © 2025 Leith, Lye, Delaney, Mclenachan, Chen, Atlas and Wong. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Elaine Y. Wong, Ear Science Institute Australia, Subiaco, Australia
Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.