Delivery is key: lessons learnt from developing splice‐switching antisense therapies
Abstract 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. The main obstacle impeding the clinical translation of this approac...
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| Format: | Article |
| Language: | English |
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Springer Nature
2017-03-01
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| Series: | EMBO Molecular Medicine |
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| Online Access: | https://doi.org/10.15252/emmm.201607199 |
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| author | 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 |
| author_facet | 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 |
| author_sort | Caroline Godfrey |
| collection | DOAJ |
| description | Abstract 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. The main obstacle impeding the clinical translation of this approach is the relatively poor delivery of antisense oligonucleotides to target tissues after systemic delivery. We are a group of researchers closely involved in the development of these therapies and would like to communicate our discussions concerning the validity of standard methodologies currently used in their pre‐clinical development, the gaps in current knowledge and the pertinent challenges facing the field. We therefore make recommendations in order to focus future research efforts and facilitate a wider application of therapeutic antisense oligonucleotides. |
| format | Article |
| id | doaj-art-dc24c7db81c9430cadef6dc5077b7915 |
| institution | DOAJ |
| issn | 1757-4676 1757-4684 |
| language | English |
| publishDate | 2017-03-01 |
| publisher | Springer Nature |
| record_format | Article |
| series | EMBO Molecular Medicine |
| spelling | doaj-art-dc24c7db81c9430cadef6dc5077b79152025-08-20T03:06:00ZengSpringer NatureEMBO Molecular Medicine1757-46761757-46842017-03-019554555710.15252/emmm.201607199Delivery is key: lessons learnt from developing splice‐switching antisense therapiesCaroline Godfrey0Lourdes R Desviat1Bård Smedsrød2France Piétri‐Rouxel3Michela A Denti4Petra Disterer5Stéphanie Lorain6Gisela Nogales‐Gadea7Valentina Sardone8Rayan Anwar9Samir EL Andaloussi10Taavi Lehto11Bernard Khoo12Camilla Brolin13Willeke MC van Roon‐Mom14Aurélie Goyenvalle15Annemieke Aartsma‐Rus16Virginia Arechavala‐Gomeza17Department of Physiology, Anatomy and Genetics, University of OxfordCentro de Biología Molecular Severo Ochoa UAM‐CSIC, CIBERER, IdiPaz, Universidad Autónoma de MadridDepartment of Medical Biology, University of TromsøUPMC, INSERM, UMRS 974, CNRS FRE 3617, Institut de MyologieCentre for Integrative Biology, University of TrentoCentre for Amyloidosis and Acute Phase Proteins, Division of Medicine, University College LondonUPMC, INSERM, UMRS 974, CNRS FRE 3617, Institut de MyologieGrup d'Investigació en Malalties Neuromusculars i Neuropediatriques, Institut d' Investigació en Ciències de la Salut Germans Trias i Pujol, BadalonaDubowitz Neuromuscular Centre and Developmental Neuroscience Programme, Institute of Child Health, University College LondonDrug Discovery Informatics Lab, Qasemi‐Research Center, Al‐Qasemi Academic CollegeDepartment of Physiology, Anatomy and Genetics, University of OxfordDepartment of Laboratory Medicine, Karolinska InstituteCentre for Neuroendocrinology, Division of Medicine, University College LondonDepartment of Cellular and Molecular Medicine, University of CopenhagenDepartment of Human Genetics, Leiden University Medical CenterINSERM U1179, UFR des sciences de la santé, Université Versailles Saint QuentinDepartment of Human Genetics, Leiden University Medical CenterNeuromuscular Disorders Group, BioCruces Health Research Institute, BarakaldoAbstract 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. The main obstacle impeding the clinical translation of this approach is the relatively poor delivery of antisense oligonucleotides to target tissues after systemic delivery. We are a group of researchers closely involved in the development of these therapies and would like to communicate our discussions concerning the validity of standard methodologies currently used in their pre‐clinical development, the gaps in current knowledge and the pertinent challenges facing the field. We therefore make recommendations in order to focus future research efforts and facilitate a wider application of therapeutic antisense oligonucleotides.https://doi.org/10.15252/emmm.201607199antisense oligonucleotidesdeliverypre‐clinical modelsRNA therapytoxicity |
| spellingShingle | 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 Delivery is key: lessons learnt from developing splice‐switching antisense therapies EMBO Molecular Medicine antisense oligonucleotides delivery pre‐clinical models RNA therapy toxicity |
| title | Delivery is key: lessons learnt from developing splice‐switching antisense therapies |
| title_full | Delivery is key: lessons learnt from developing splice‐switching antisense therapies |
| title_fullStr | Delivery is key: lessons learnt from developing splice‐switching antisense therapies |
| title_full_unstemmed | Delivery is key: lessons learnt from developing splice‐switching antisense therapies |
| title_short | Delivery is key: lessons learnt from developing splice‐switching antisense therapies |
| title_sort | delivery is key lessons learnt from developing splice switching antisense therapies |
| topic | antisense oligonucleotides delivery pre‐clinical models RNA therapy toxicity |
| url | https://doi.org/10.15252/emmm.201607199 |
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