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

Author:

Godfrey Caroline1,Desviat Lourdes R2,Smedsrød Bård3,Piétri‐Rouxel France4,Denti Michela A5,Disterer Petra6,Lorain Stéphanie4,Nogales‐Gadea Gisela7,Sardone Valentina8,Anwar Rayan910,EL Andaloussi Samir111,Lehto Taavi1112,Khoo Bernard13,Brolin Camilla14,Roon‐Mom Willeke MC15,Goyenvalle Aurélie16,Aartsma‐Rus Annemieke15,Arechavala‐Gomeza Virginia17ORCID

Affiliation:

1. Department of Physiology, Anatomy and Genetics University of Oxford Oxford UK

2. Centro de Biología Molecular Severo Ochoa UAM‐CSIC CIBERER IdiPaz Universidad Autónoma de Madrid Madrid Spain

3. Department of Medical Biology University of Tromsø Tromsø Norway

4. UPMC, INSERM, UMRS 974 CNRS FRE 3617 Institut de Myologie Paris France

5. Centre for Integrative Biology University of Trento Trento Italy

6. Centre for Amyloidosis and Acute Phase Proteins Division of Medicine University College London London UK

7. Grup d'Investigació en Malalties Neuromusculars i Neuropediatriques Institut d' Investigació en Ciències de la Salut Germans Trias i Pujol Badalona Barcelona Spain

8. Dubowitz Neuromuscular Centre and Developmental Neuroscience Programme Institute of Child Health University College London London UK

9. Drug Discovery Informatics Lab Qasemi‐Research Center Al‐Qasemi Academic College Baka El‐Garbiah Israel

10. Drug Discovery and Development Laboratory Institute of Applied Research Galilee Society Shefa‐Amr Israel

11. Department of Laboratory Medicine Karolinska Institute Stockholm Sweden

12. Institute of Technology University of Tartu Tartu Estonia

13. Centre for Neuroendocrinology Division of Medicine University College London London UK

14. Department of Cellular and Molecular Medicine University of Copenhagen Copenhagen Denmark

15. Department of Human Genetics Leiden University Medical Center Leiden The Netherlands

16. INSERM U1179 UFR des sciences de la santé Université Versailles Saint Quentin Montigny‐le‐Bretonneux France

17. Neuromuscular Disorders Group BioCruces Health Research Institute Barakaldo Bizkaia Spain

Funder

Ministerio de Economía y Competitividad

European Cooperation in Science and Technology

Duchenne Parent Project

Instituto de Salud Carlos III

European Regional Development Fund

European Commission

Seventh Framework Programme

Ministry of Economy

Svenska Forskningsrådet Formas

Svenska Sällskapet för Medicinsk Forskning

Eesti Teadusagentuur

Wellcome Trust

Medical Research Council

British Heart Foundation

Det Frie Forskningsråd

ZonMw

Hersenstichting

AFM-Téléthon

Institut National de la Santé et de la Recherche Médicale

Agence Nationale de la Recherche

Fundación Ramón Areces

Prinses Beatrix Spierfonds

Fondazione Telethon

Ministero della Salute

Ministry of Science, Technology and Space

Publisher

EMBO

Subject

Molecular Medicine

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