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Antisense Oligonucleotide- and CRISPR-Cas9-Mediated Rescue of mRNA Splicing for a Deep Intronic CLRN1 Mutation.
Panagiotopoulos, Anna-Lena; Karguth, Nina; Pavlou, Marina; Böhm, Sybille; Gasparoni, Gilles; Walter, Jörn; Graf, Alexander; Blum, Helmut; Biel, Martin; Riedmayr, Lisa Maria; Becirovic, Elvir.
Afiliación
  • Panagiotopoulos AL; Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität München, Munich, Germany.
  • Karguth N; Center for Integrated Protein Science Munich CIPSM, Munich, Germany; Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität München, Munich, Germany.
  • Pavlou M; Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität München, Munich, Germany; Department of Ophthalmology, Ludwig-Maximilians-Universität München, Munich, Germany.
  • Böhm S; Center for Integrated Protein Science Munich CIPSM, Munich, Germany; Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität München, Munich, Germany.
  • Gasparoni G; Department of Genetics, Saarland University, Saarbrücken, Germany.
  • Walter J; Department of Genetics, Saarland University, Saarbrücken, Germany.
  • Graf A; Gene Center Munich, Ludwig-Maximilians-Universität München, Munich, Germany.
  • Blum H; Gene Center Munich, Ludwig-Maximilians-Universität München, Munich, Germany.
  • Biel M; Center for Integrated Protein Science Munich CIPSM, Munich, Germany; Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität München, Munich, Germany.
  • Riedmayr LM; 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.
  • Becirovic E; 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.
Mol Ther Nucleic Acids ; 21: 1050-1061, 2020 Sep 04.
Article en En | MEDLINE | ID: mdl-32841912
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Idioma: En Revista: Mol Ther Nucleic Acids Año: 2020 Tipo del documento: Article País de afiliación: Alemania
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Idioma: En Revista: Mol Ther Nucleic Acids Año: 2020 Tipo del documento: Article País de afiliación: Alemania