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

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Title: Delivery is key: lessons learnt from developing splice-switching antisense therapies.
Authors: Godfrey C; Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK., Desviat LR; Centro de Biología Molecular Severo Ochoa UAM-CSIC, CIBERER, IdiPaz, Universidad Autónoma de Madrid, Madrid, Spain., Smedsrød B; Department of Medical Biology, University of Tromsø, Tromsø, Norway., Piétri-Rouxel F; UPMC, INSERM, UMRS 974, CNRS FRE 3617, Institut de Myologie, Paris, France., Denti MA; Centre for Integrative Biology, University of Trento, Trento, Italy., Disterer P; Centre for Amyloidosis and Acute Phase Proteins, Division of Medicine, University College London, London, UK., Lorain S; UPMC, INSERM, UMRS 974, CNRS FRE 3617, Institut de Myologie, Paris, France., Nogales-Gadea G; Grup d'Investigació en Malalties Neuromusculars i Neuropediatriques, Institut d' Investigació en Ciències de la Salut Germans Trias i Pujol, Badalona Barcelona, Spain., Sardone V; Dubowitz Neuromuscular Centre and Developmental Neuroscience Programme, Institute of Child Health, University College London, London, UK., Anwar R; Drug Discovery Informatics Lab, Qasemi-Research Center, Al-Qasemi Academic College, Baka El-Garbiah, Israel.; Drug Discovery and Development Laboratory, Institute of Applied Research, Galilee Society, Shefa-Amr, Israel., El Andaloussi S; Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK.; Department of Laboratory Medicine, Karolinska Institute, Stockholm, Sweden., Lehto T; Department of Laboratory Medicine, Karolinska Institute, Stockholm, Sweden.; Institute of Technology, University of Tartu, Tartu, Estonia., Khoo B; Centre for Neuroendocrinology, Division of Medicine, University College London, London, UK., Brolin C; Department of Cellular and Molecular Medicine, University of Copenhagen, Copenhagen, Denmark., van Roon-Mom WM; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands., Goyenvalle A; INSERM U1179, UFR des sciences de la santé, Université Versailles Saint Quentin, Montigny-le-Bretonneux, France., Aartsma-Rus A; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands., Arechavala-Gomeza V; Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo Bizkaia, Spain virginia.arechavalagomeza@osakidetza.eus.
Source: EMBO molecular medicine [EMBO Mol Med] 2017 May; Vol. 9 (5), pp. 545-557.
Publication Type: Journal Article; Review; Research Support, Non-U.S. Gov't
Journal Info: Publisher: EMBO Press Country of Publication: Germany NLM ID: 101487380 Publication Model: Print Cited Medium: Internet ISSN: 1757-4684 (Electronic) Linking ISSN: 17574676 NLM ISO Abbreviation: EMBO Mol Med Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Delivery is key: lessons learnt from developing splice-switching antisense therapies.
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  Data: <searchLink fieldCode="AU" term="%22Godfrey+C%22">Godfrey C</searchLink>; Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK.<br /><searchLink fieldCode="AU" term="%22Desviat+LR%22">Desviat LR</searchLink>; Centro de Biología Molecular Severo Ochoa UAM-CSIC, CIBERER, IdiPaz, Universidad Autónoma de Madrid, Madrid, Spain.<br /><searchLink fieldCode="AU" term="%22Smedsrød+B%22">Smedsrød B</searchLink>; Department of Medical Biology, University of Tromsø, Tromsø, Norway.<br /><searchLink fieldCode="AU" term="%22Piétri-Rouxel+F%22">Piétri-Rouxel F</searchLink>; UPMC, INSERM, UMRS 974, CNRS FRE 3617, Institut de Myologie, Paris, France.<br /><searchLink fieldCode="AU" term="%22Denti+MA%22">Denti MA</searchLink>; Centre for Integrative Biology, University of Trento, Trento, Italy.<br /><searchLink fieldCode="AU" term="%22Disterer+P%22">Disterer P</searchLink>; Centre for Amyloidosis and Acute Phase Proteins, Division of Medicine, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Lorain+S%22">Lorain S</searchLink>; UPMC, INSERM, UMRS 974, CNRS FRE 3617, Institut de Myologie, Paris, France.<br /><searchLink fieldCode="AU" term="%22Nogales-Gadea+G%22">Nogales-Gadea G</searchLink>; Grup d'Investigació en Malalties Neuromusculars i Neuropediatriques, Institut d' Investigació en Ciències de la Salut Germans Trias i Pujol, Badalona Barcelona, Spain.<br /><searchLink fieldCode="AU" term="%22Sardone+V%22">Sardone V</searchLink>; Dubowitz Neuromuscular Centre and Developmental Neuroscience Programme, Institute of Child Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Anwar+R%22">Anwar R</searchLink>; Drug Discovery Informatics Lab, Qasemi-Research Center, Al-Qasemi Academic College, Baka El-Garbiah, Israel.; Drug Discovery and Development Laboratory, Institute of Applied Research, Galilee Society, Shefa-Amr, Israel.<br /><searchLink fieldCode="AU" term="%22El+Andaloussi+S%22">El Andaloussi S</searchLink>; Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK.; Department of Laboratory Medicine, Karolinska Institute, Stockholm, Sweden.<br /><searchLink fieldCode="AU" term="%22Lehto+T%22">Lehto T</searchLink>; Department of Laboratory Medicine, Karolinska Institute, Stockholm, Sweden.; Institute of Technology, University of Tartu, Tartu, Estonia.<br /><searchLink fieldCode="AU" term="%22Khoo+B%22">Khoo B</searchLink>; Centre for Neuroendocrinology, Division of Medicine, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Brolin+C%22">Brolin C</searchLink>; Department of Cellular and Molecular Medicine, University of Copenhagen, Copenhagen, Denmark.<br /><searchLink fieldCode="AU" term="%22van+Roon-Mom+WM%22">van Roon-Mom WM</searchLink>; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Goyenvalle+A%22">Goyenvalle A</searchLink>; INSERM U1179, UFR des sciences de la santé, Université Versailles Saint Quentin, Montigny-le-Bretonneux, France.<br /><searchLink fieldCode="AU" term="%22Aartsma-Rus+A%22">Aartsma-Rus A</searchLink>; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Arechavala-Gomeza+V%22">Arechavala-Gomeza V</searchLink>; Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo Bizkaia, Spain virginia.arechavalagomeza@osakidetza.eus.
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