Publication: Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations
Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations
Date
Date
Date
Citations
Dulla, K., Slijkerman, R., van Diepen, H. C., Albert, S., Dona, M., et al, Zang, J., & Neuhauss, S. C. F. (2021). Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations. Molecular Therapy, 29(8), 2441–2455. https://doi.org/10.1016/j.ymthe.2021.04.024
Abstract
Abstract
Abstract
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$^{rmc1}$ mutants resulted in the production of usherinΔexon 13 protein and a completely resto
Additional indexing
Creators (Authors)
- Dulla, Kalyan
- Slijkerman, Ralph
- van Diepen, Hester C
- Albert, Silvia
- Dona, Margo
- Zang, Jingjing
Volume
Volume
Volume
Number
Number
Number
Page range/Item number
Page range/Item number
Page range/Item number
Page end
Page end
Page end
Item Type
Item Type
Item Type
In collections
Language
Language
Language
Publication date
Publication date
Publication date
Date available
Date available
Date available
ISSN or e-ISSN
ISSN or e-ISSN
ISSN or e-ISSN
OA Status
OA Status
OA Status
Free Access at
Free Access at
Free Access at
Publisher DOI
Citations
Dulla, K., Slijkerman, R., van Diepen, H. C., Albert, S., Dona, M., et al, Zang, J., & Neuhauss, S. C. F. (2021). Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations. Molecular Therapy, 29(8), 2441–2455. https://doi.org/10.1016/j.ymthe.2021.04.024
