<i>USH2A</i> -Mutated Human Retinal Organoids Model Rod–Cone Dystrophy
Our findings suggest that molecular changes precede overt photoreceptor loss in USH2A RP, and pathogenesis may begin before clinical symptoms emerge. By defining early and late disease features, we provide new insight on the developmental origins of USH2A RP to guide therapeutic strategies.
Authors
- Brian G. Ballios (ORCID: https://orcid.org/0000-0002-4644-3339)
- Cassandra D’Amata
- Kristen E. Ashworth (ORCID: https://orcid.org/0000-0002-4939-021X)
- Élise Héon
- Jiajie Zhang (ORCID: https://orcid.org/0009-0007-2455-8315)
Institutions
- Twitter (United States) (US)
- University of Toronto (CA)
- Hospital for Sick Children (CA)
- Krembil Foundation (CA)
- Krembil Research Institute
Publication Details
- Journal
- Investigative Ophthalmology & Visual Science
- Published
- 2025-11-03
- DOI
- https://doi.org/10.1167/iovs.66.14.2
- Primary Topic
- Retinal Development and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00