Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties

Molecular Therapy - Nucleic Acids - Tập 29 - Trang 955-968 - 2022
Katarzyna Chwalenia1,2, Jacopo Oieni1, Joanna Zemła3, Małgorzata Lekka3, Nina Ahlskog1,2, Anna M.L. Coenen-Stass4, Graham McClorey1, Matthew J.A. Wood1,2,5, Yulia Lomonosova1,2,5, Thomas C. Roberts1,2,5
1Department of Paediatrics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK
2Institute of Developmental and Regenerative Medicine, University of Oxford, IMS-Tetsuya Nakamura Building, Old Road Campus, Roosevelt Drive, Headington, Oxford OX3 7TY, UK
3Department of Biophysical Microstructures, Institute of Nuclear Physics, Polish Academy of Sciences, PL-31342 Kraków, Poland
4Department of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK
5MDUK Oxford Neuromuscular Centre, South Parks Road, Oxford OX1 3QX, UK

Tài liệu tham khảo

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