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

Open Access

Review

Author Affiliations

Caroline Godfrey¹,
Lourdes R Desviat²,
Bård Smedsrød³,
France Piétri‐Rouxel⁴,
Michela A Denti⁵,
Petra Disterer⁶,
Stéphanie Lorain⁴,
Gisela Nogales‐Gadea⁷,
Valentina Sardone⁸,
Rayan Anwar⁹,¹⁰,
Samir EL Andaloussi¹,¹¹,
Taavi Lehto¹¹,¹²,
Bernard Khoo¹³,
Camilla Brolin¹⁴,
Willeke MC van Roon‐Mom¹⁵,
Aurélie Goyenvalle¹⁶,
Annemieke Aartsma‐Rus¹⁵ and
Virginia Arechavala‐Gomeza (virginia.arechavalagomeza{at}osakidetza.eus)*,¹⁷

¹Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK
²Centro de Biología Molecular Severo Ochoa UAM‐CSIC, CIBERER, IdiPaz, Universidad Autónoma de Madrid, Madrid, Spain
³Department of Medical Biology, University of Tromsø, Tromsø, Norway
⁴UPMC, INSERM, UMRS 974, CNRS FRE 3617, Institut de Myologie, Paris, France
⁵Centre for Integrative Biology, University of Trento, Trento, Italy
⁶Centre for Amyloidosis and Acute Phase Proteins, Division of Medicine, University College London, London, UK
⁷Grup d'Investigació en Malalties Neuromusculars i Neuropediatriques, Institut d' Investigació en Ciències de la Salut Germans Trias i Pujol, Badalona Barcelona, Spain
⁸Dubowitz Neuromuscular Centre and Developmental Neuroscience Programme, Institute of Child Health, University College London, London, UK
⁹Drug Discovery Informatics Lab, Qasemi‐Research Center, Al‐Qasemi Academic College, Baka El‐Garbiah, Israel
¹⁰Drug Discovery and Development Laboratory, Institute of Applied Research, Galilee Society, Shefa‐Amr, Israel
¹¹Department of Laboratory Medicine, Karolinska Institute, Stockholm, Sweden
¹²Institute of Technology, University of Tartu, Tartu, Estonia
¹³Centre for Neuroendocrinology, Division of Medicine, University College London, London, UK
¹⁴Department of Cellular and Molecular Medicine, University of Copenhagen, Copenhagen, Denmark
¹⁵Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands
¹⁶INSERM U1179, UFR des sciences de la santé, Université Versailles Saint Quentin, Montigny‐le‐Bretonneux, France
¹⁷Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo Bizkaia, Spain