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13 p, 1.6 MB Delivery is key: lessons learnt from developing splice-switching antisense therapies / Godfrey, Caroline (University of Oxford. Department of Physiology, Anatomy and Genetics) ; Desviat, Lourdes R (Centro de Biología Molecular Severo Ochoa UAM-CSIC) ; Smedsrød, Bård (University of TromsØ. Department of Medical Biology) ; Piétri-Rouxel, France (Institut de Myologie, Paris) ; Denti, Michela A (Università di Trento. Centre for Integrative Biology) ; Disterer, Petra (University College London. Centre for Amyloidosis and Acute Phase Proteins) ; Lorain, Stéphanie (Institut de Myologie, Paris) ; Nogales Gadea, Gisela (Institut Germans Trias i Pujol. Grup d'Investigació en Malalties Neuromuscular i Neuropediàtriques) ; Sardone, Valentina (University College London) ; Anwar, Rayan (Drug Discovery Informatics Lap, Qasemi-Research Center, Al-Qasemi Academic College) ; EL Andaloussi, Samir (Karolinska Institute. Department of Laboratory Medicine) ; Lehto, Taavi (University of Tartu. Institute of Technology) ; Khoo, Bernard (University College London. Centre for Neuroendocrinology) ; Brolin, Camilla (University of Copenhagen. Department of Cellular and Molecular Medicine) ; van Roon-Mom, Willeke MC (Leiden University Medical Center. DEpartment of Human Genetics) ; Goyenvalle, Aurélie (Université Versailles Saint Quentin) ; Aartsma-Rus, Annemieke (Leiden University Medical Center. Department of Human Genetics) ; Arechavala-Gomeza, Virginia (Neuromuscular Disorders Group. BioCruces Health Research Insitute, Bizkaia) ; Universitat Autònoma de Barcelona
The use of splice-switching antisense therapy is highly promising, with a wealth of pre-clinical data and numerous clinical trials ongoing. Nevertheless, its potential to treat a variety of disorders has yet to be realized. [...]
2017 - 10.15252/emmm.201607199
EMBO Molecular Medicine, Vol. 9 Núm. 5 (2017) , p. 545-557  

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1 Aartsma-Rus, Annemieke
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