Delivery of oligonucleotide‐based therapeutics: challenges and opportunities-Reference-Cited by-同舟云学术

Delivery of oligonucleotide‐based therapeutics: challenges and opportunities

Published:2021-04-06 Issue:4 Volume:13 Page:
ISSN:1757-4676
Container-title:EMBO Molecular Medicine
language:en
Short-container-title:EMBO Mol Med

Author:

Hammond Suzan M¹^ORCID,Aartsma‐Rus Annemieke²^ORCID,Alves Sandra³^ORCID,Borgos Sven E⁴^ORCID,Buijsen Ronald A M²^ORCID,Collin Rob W J⁵^ORCID,Covello Giuseppina⁶⁷^ORCID,Denti Michela A⁷^ORCID,Desviat Lourdes R⁸^ORCID,Echevarría Lucía⁹^ORCID,Foged Camilla¹⁰^ORCID,Gaina Gisela¹¹¹²^ORCID,Garanto Alejandro⁵¹³^ORCID,Goyenvalle Aurelie T¹⁴^ORCID,Guzowska Magdalena¹⁵,Holodnuka Irina¹⁶^ORCID,Jones David R¹⁷,Krause Sabine¹⁸^ORCID,Lehto Taavi¹⁹²⁰^ORCID,Montolio Marisol²¹²²^ORCID,Van Roon‐Mom Willeke²^ORCID,Arechavala‐Gomeza Virginia²³²⁴^ORCID

Affiliation:

1. Department of Paediatrics University of Oxford Oxford UK

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

3. Department of Human Genetics, Research and Development Unit National Health Institute Doutor Ricardo Jorge Porto Portugal

4. Department of Biotechnology and Nanomedicine SINTEF AS Trondheim Norway

5. Department of Human Genetics and Donders Institute for Brain, Cognition and Behaviour Radboud University Medical Center Nijmegen The Netherlands

6. Department of Biology University of Padova Padova Italy

7. Department of Cellular, Computational and Integrative Biology ‐ CIBIO University of Trento Trento Italy

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

9. SQY Therapeutics Montigny‐le‐Bretonneux France

10. Department of Pharmacy Faculty of Health and Medical Sciences University of Copenhagen Copenhagen Ø Denmark

11. Victor Babes National Institute of Pathology Bucharest Romania

12. Department of Biochemistry and Molecular Biology University of Bucharest Bucharest Romania

13. Department of Pediatrics Radboud University Medical Center Nijmegen The Netherlands

14. Université Paris‐Saclay, UVSQ, Inserm, END‐ICAP Versailles France

15. Department of Physiological Sciences Faculty of Veterinary Medicine Warsaw University of Life Sciences – SGGW Warsaw Poland

16. Institute of Microbiology and Virology Riga Stradins University Riga Latvia

17. MHRA 10 South Colonnade London UK

18. Department of Neurology Friedrich‐Baur‐Institute Ludwig‐Maximilians‐University of Munich Munich Germany

19. Institute of Technology University of Tartu Tartu Estonia

20. Division of Biomolecular and Cellular Medicine Department of Laboratory Medicine Karolinska Institutet Huddinge Sweden

21. Duchenne Parent Project España Madrid Spain

22. Department of Cell Biology, Fisiology and Immunology Faculty of Biology University of Barcelona Barcelona Spain

23. Neuromuscular Disorders Group Biocruces Bizkaia Health Research Institute Barakaldo Spain

24. Ikerbasque, Basque Foundation for Science Bilbao Spain

Funder

Ikerbasque, Basque Foundation for Science

Duchenne Parent Project

Stichting Retina Fonds

Ministerio de Ciencia e Innovación

Eesti Teadusagentuur

Muscular Dystrophy UK

Publisher

EMBO

Subject

Molecular Medicine

Link

https://onlinelibrary.wiley.com/doi/pdf/10.15252/emmm.202013243

Reference219 articles.

1. FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides

2. Why dystrophin quantification is key in the eteplirsen saga

3. The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy

4. Merging organoid and organ-on-a-chip technology to generate complex multi-layer tissue models in a human retina-on-a-chip platform

5. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

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