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Kalyan Dulla, Ralph Slijkerman, Hester C van Diepen +22 more · 2021 · Molecular therapy : the journal of the American Society of Gene Therapy · Elsevier · added 2026-04-24
Mutations in USH2A are among the most common causes of syndromic and non-syndromic retinitis pigmentosa (RP). The two most recurrent mutations in USH2A, c.2299delG and c.2276G > T, both reside in exon Show more
Mutations in USH2A are among the most common causes of syndromic and non-syndromic retinitis pigmentosa (RP). The two most recurrent mutations in USH2A, c.2299delG and c.2276G > T, both reside in exon 13. Skipping exon 13 from the USH2A transcript presents a potential treatment modality in which the resulting transcript is predicted to encode a slightly shortened usherin protein. Morpholino-induced skipping of ush2a exon 13 in zebrafish ush2a Show less
no PDF DOI: 10.1016/j.ymthe.2021.04.024
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