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Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties.
Chwalenia, Katarzyna; Oieni, Jacopo; Zemla, Joanna; Lekka, Malgorzata; Ahlskog, Nina; Coenen-Stass, Anna M L; McClorey, Graham; Wood, Matthew J A; Lomonosova, Yulia; Roberts, Thomas C.
Affiliation
  • Chwalenia K; Department of Paediatrics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK.
  • Oieni J; Institute of Developmental and Regenerative Medicine, University of Oxford, IMS-Tetsuya Nakamura Building, Old Road Campus, Roosevelt Drive, Headington, Oxford OX3 7TY, UK.
  • Zemla J; Department of Paediatrics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK.
  • Lekka M; Department of Biophysical Microstructures, Institute of Nuclear Physics, Polish Academy of Sciences, PL-31342 Kraków, Poland.
  • Ahlskog N; Department of Biophysical Microstructures, Institute of Nuclear Physics, Polish Academy of Sciences, PL-31342 Kraków, Poland.
  • Coenen-Stass AML; Department of Paediatrics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK.
  • McClorey G; Institute of Developmental and Regenerative Medicine, University of Oxford, IMS-Tetsuya Nakamura Building, Old Road Campus, Roosevelt Drive, Headington, Oxford OX3 7TY, UK.
  • Wood MJA; Department of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK.
  • Lomonosova Y; Department of Paediatrics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK.
  • Roberts TC; Department of Paediatrics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK.
Mol Ther Nucleic Acids ; 29: 955-968, 2022 Sep 13.
Article in En | MEDLINE | ID: mdl-36159597
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Ther Nucleic Acids Year: 2022 Type: Article Affiliation country: United kingdom
Fulltext
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PubMed Links
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Ther Nucleic Acids Year: 2022 Type: Article Affiliation country: United kingdom