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Mini-Pcdh15b Gene Therapy Rescues Visual Deficits in a Zebrafish Model of Usher Syndrome Type 1F

2025-11-06

Abstract excerpt

<h4>ABSTRACT</h4> Usher syndrome type 1F (USH1F) is a severe inherited disorder caused by mutations in PCDH15 , resulting in congenital deafness, vestibular dysfunction, and progressive retinal degeneration leading to blindness. While cochlear implantation can restore hearing, no therapeutic interventions currently exist for vision loss. Gene augmentation therapy represents a promising approach; however, the PC...

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Literature Corpus work
c4c7eb62-3cbd-5223-9da6-a995b0e70e40
DOI
10.1101/2025.11.05.686814
Open publication

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