A protein domain-oriented approach to expand the opportunities of therapeutic exon skipping for USH2A-associated retinitis pigmentosa

Loss-of-function mutations in USH2A are among the most common causes of syndromic and non-syndromic retinitis pigmentosa (RP). We previously presented skipping of USH2A exon 13 as a promising treatment paradigm for USH2A-associated RP. However, RP-associated mutations are often private, and evenly d...

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Detalles Bibliográficos
Autores principales: Schellens, Renske T.W., Broekman, Sanne, Peters, Theo, Graave, Pam, Malinar, Lucija, Venselaar, Hanka, Kremer, Hannie, De Vrieze, Erik, Van Wijk, Erwin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10258241/
https://www.ncbi.nlm.nih.gov/pubmed/37313440
http://dx.doi.org/10.1016/j.omtn.2023.05.020

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