Antisense Oligonucleotide- and CRISPR-Cas9-Mediated Rescue of mRNA Splicing for a Deep Intronic CLRN1 Mutation

Mutations in CLRN1 cause Usher syndrome (USH) type III (USH3A), a disease characterized by progressive hearing impairment, retinitis pigmentosa, and vestibular dysfunction. Due to the lack of appropriate disease models, no efficient therapy for retinitis pigmentosa in USH patients exists so far. In...

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Detalles Bibliográficos
Autores principales: Panagiotopoulos, Anna-Lena, Karguth, Nina, Pavlou, Marina, Böhm, Sybille, Gasparoni, Gilles, Walter, Jörn, Graf, Alexander, Blum, Helmut, Biel, Martin, Riedmayr, Lisa Maria, Becirovic, Elvir
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7452116/
https://www.ncbi.nlm.nih.gov/pubmed/32841912
http://dx.doi.org/10.1016/j.omtn.2020.07.036

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