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Delivery is key: lessons learnt from developing splice‐switching antisense therapies

Caroline Godfrey, Lourdes R Desviat, Bård Smedsrød, France Piétri‐Rouxel, Michela A Denti, Petra Disterer, Stéphanie Lorain, Gisela Nogales‐Gadea, Valentina Sardone, Rayan Anwar, Samir EL Andaloussi, Taavi Lehto, Bernard Khoo, Camilla Brolin, Willeke MC van Roon‐Mom, Aurélie Goyenvalle, Annemieke Aartsma‐Rus, Virginia Arechavala‐Gomeza
DOI 10.15252/emmm.201607199 | Published online 13.03.2017
EMBO Molecular Medicine (2017) e201607199

Author Affiliations

  1. Caroline Godfrey1,
  2. Lourdes R Desviat2,
  3. Bård Smedsrød3,
  4. France Piétri‐Rouxel4,
  5. Michela A Denti5,
  6. Petra Disterer6,
  7. Stéphanie Lorain4,
  8. Gisela Nogales‐Gadea7,
  9. Valentina Sardone8,
  10. Rayan Anwar9,10,
  11. Samir EL Andaloussi1,11,
  12. Taavi Lehto11,12,
  13. Bernard Khoo13,
  14. Camilla Brolin14,
  15. Willeke MC van Roon‐Mom15,
  16. Aurélie Goyenvalle16,
  17. Annemieke Aartsma‐Rus15 and
  18. Virginia Arechavala‐Gomeza (virginia.arechavalagomeza{at}osakidetza.eus)*,17
  1. 1Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK
  2. 2Centro de Biología Molecular Severo Ochoa UAM‐CSIC, CIBERER, IdiPaz, Universidad Autónoma de Madrid, Madrid, Spain
  3. 3Department of Medical Biology, University of Tromsø, Tromsø, Norway
  4. 4UPMC, INSERM, UMRS 974, CNRS FRE 3617, Institut de Myologie, Paris, France
  5. 5Centre for Integrative Biology, University of Trento, Trento, Italy
  6. 6Centre for Amyloidosis and Acute Phase Proteins, Division of Medicine, University College London, London, UK
  7. 7Grup d'Investigació en Malalties Neuromusculars i Neuropediatriques, Institut d' Investigació en Ciències de la Salut Germans Trias i Pujol, Badalona Barcelona, Spain
  8. 8Dubowitz Neuromuscular Centre and Developmental Neuroscience Programme, Institute of Child Health, University College London, London, UK
  9. 9Drug Discovery Informatics Lab, Qasemi‐Research Center, Al‐Qasemi Academic College, Baka El‐Garbiah, Israel
  10. 10Drug Discovery and Development Laboratory, Institute of Applied Research, Galilee Society, Shefa‐Amr, Israel
  11. 11Department of Laboratory Medicine, Karolinska Institute, Stockholm, Sweden
  12. 12Institute of Technology, University of Tartu, Tartu, Estonia
  13. 13Centre for Neuroendocrinology, Division of Medicine, University College London, London, UK
  14. 14Department of Cellular and Molecular Medicine, University of Copenhagen, Copenhagen, Denmark
  15. 15Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands
  16. 16INSERM U1179, UFR des sciences de la santé, Université Versailles Saint Quentin, Montigny‐le‐Bretonneux, France
  17. 17Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo Bizkaia, Spain
  1. ↵*Corresponding author. Tel: +34 946007967; E‐mail: virginia.arechavalagomeza{at}osakidetza.eus
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Volume 10, Issue 4
01 April 2018 | pp -
EMBO Molecular Medicine: 10 (4)
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