Article

Gene Supplementation of <i>MYO7A</i> or activation of <i>Myo7b</i> for treatment of Usher syndrome 1B

2026-07-03

Abstract excerpt

Mutations in MYO7A result in the most severe subtype of Usher syndrome, the leading genetic cause of deafblindness. The large size of MYO7A requires dual adeno-associated virus (AAV) vectors for gene transfer or alternative methods to treat retinal defects. Here, we evaluated two treatment approaches: i) Supplementation of the human MYO7A gene via dual mRNA trans-splicing AAVs, and ii) CRISPR/Cas-mediated activ...

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Literature Corpus work: f54f4c68-0731-50c8-9d2a-adc4d8111b25
DOI: 10.64898/2026.07.02.736025

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