Article

Expanding the options for therapeutic exon skipping as a future treatment for <i>USH2A</i> -associated disease by 3D structural modeling of newly formed hybrid domains

2026-04-28

Abstract excerpt

<h4>ABSTRACT</h4> Usher syndrome, the leading cause of hereditary deaf-blindness affecting approximately 1 in 15,000 individuals worldwide, is currently still untreatable. Antisense oligonucleotide-based exon skipping has shown significant therapeutic promise for USH2A -associated retinal dysfunction. Selection of (combinations of) exons suitable for therapeutic exon skipping within the fibronectin type 3 (FN3)...

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Literature Corpus work: eb06d78d-7dc0-5038-99ab-6a657d19c3f7
DOI: 10.64898/2026.04.24.720583

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