Exon 13-skipped USH2A protein retains functional integrity in mice, suggesting an exo-skipping therapeutic approach to treat USH2A-associated disease
Nachiket Pendse(Massachusetts Eye and Ear Infirmary), Qin Liu(Massachusetts Eye and Ear Infirmary), Sebastian Gloskowski(Editas Medicine (United States)), Morgan L. Maeder(Editas Medicine (United States)), Zheng‐Yi Chen(Massachusetts Eye and Ear Infirmary), Verónica Lamas(Massachusetts Eye and Ear Infirmary), Basil S. Pawlyk(Berman Center for Outcomes and Clinical Research), Eric A. Pierce(Broad Institute)
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