Article

A Genomic Analysis of Usher Syndrome: Population-Scale Prevalence and Therapeutic Targets

2025-02-27

Abstract excerpt

<h4>ABSTRACT</h4> Usher syndrome (USH), the most common form of deaf-blindness, displays extensive genetic, allelic, and phenotypic heterogeneity. The dual sensory impairment associated with this disorder makes Usher syndrome an important target for gene therapy, with dozens of published preclinical studies targeting multiple USH genes and using multiple gene therapy strategies. Nine genes have been conclusively l...

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Literature Corpus work: 86c81bda-11ac-5b17-8cf8-ac01a3361180
DOI: 10.1101/2025.02.27.25323008

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