Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations

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...

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Detalles Bibliográficos
Autores principales: Dulla, Kalyan, Slijkerman, Ralph, van Diepen, Hester C., Albert, Silvia, Dona, Margo, Beumer, Wouter, Turunen, Janne J., Chan, Hee Lam, Schulkens, Iris A., Vorthoren, Lars, den Besten, Cathaline, Buil, Levi, Schmidt, Iris, Miao, Jiayi, Venselaar, Hanka, Zang, Jingjing, Neuhauss, Stephan C.F., Peters, Theo, Broekman, Sanne, Pennings, Ronald, Kremer, Hannie, Platenburg, Gerard, Adamson, Peter, de Vrieze, Erik, van Wijk, Erwin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8353187/
https://www.ncbi.nlm.nih.gov/pubmed/33895329
http://dx.doi.org/10.1016/j.ymthe.2021.04.024

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