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

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第一作者： Anna-Lena,Panagiotopoulos

第一单位： Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität München, Munich, Germany.

作者： Anna-Lena,Panagiotopoulos ^[1] ; Nina,Karguth ^[2] ; Marina,Pavlou ^[3] ; Sybille,Böhm ^[2] ; Gilles,Gasparoni ^[4] ; Jörn,Walter ^[4] ; Alexander,Graf ^[5] ; Helmut,Blum ^[5] ; Martin,Biel ^[2] ; Lisa Maria,Riedmayr ^[6] ; Elvir,Becirovic ^[7]

作者单位： Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität München, Munich, Germany. ^[1] Center for Integrated Protein Science Munich CIPSM, Munich, Germany; Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität München, Munich, Germany. ^[2] Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität München, Munich, Germany; Department of Ophthalmology, Ludwig-Maximilians-Universität München, Munich, Germany. ^[3] Department of Genetics, Saarland University, Saarbrücken, Germany. ^[4] Gene Center Munich, Ludwig-Maximilians-Universität München, Munich, Germany. ^[5] Center for Integrated Protein Science Munich CIPSM, Munich, Germany; Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität München, Munich, Germany. Electronic address: lisa.riedmayr@cup.lmu.de. ^[6] Center for Integrated Protein Science Munich CIPSM, Munich, Germany; Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität München, Munich, Germany. Electronic address: elvir.becirovic@cup.lmu.de. ^[7]

关键词 AON CLRN1 CRISPR-Cas9 USH USH3 Usher syndrome antisense oligonucleotide gene editing mRNA splicing splicing mutation

DOI 10.1016/j.omtn.2020.07.036

PMID 32841912

发布时间 2024-03-29

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