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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Bibliografski detalji
Izdano u:Mol Ther Nucleic Acids
Glavni autori: Panagiotopoulos, Anna-Lena, Karguth, Nina, Pavlou, Marina, Böhm, Sybille, Gasparoni, Gilles, Walter, Jörn, Graf, Alexander, Blum, Helmut, Biel, Martin, Riedmayr, Lisa Maria, Becirovic, Elvir
Format: Artigo
Jezik:Inglês
Izdano: American Society of Gene & Cell Therapy 2020
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Article
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7452116/
https://ncbi.nlm.nih.gov/pubmed/32841912
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2020.07.036
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