Molecular BiologybioRxivPreprint
Exon 13-skipped USH2A protein retains functional integrity in mice, suggesting an exon skipping therapeutic approach to treat USH2A-associated disease.
Nachiket D. Pendse, Verónica Lamas, Morgan L. Maeder…·Qin Liu
This study shows that skipping exon 13 of the USH2A gene produces functional Usherin protein in mice, supporting exon-skipping as a therapeutic approach for USH2A-related diseases.
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