Please use this identifier to cite or link to this item: https://hdl.handle.net/20.500.12530/41247
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dc.contributor.authorGodfrey, Caroline
dc.contributor.authorDesviat, Lourdes R
dc.contributor.authorSmedsrød, Bård
dc.contributor.authorPiétri-Rouxel, France
dc.contributor.authorDenti, Michela A
dc.contributor.authorDisterer, Petra
dc.contributor.authorLorain, Stéphanie
dc.contributor.authorNogales-Gadea, Gisela
dc.contributor.authorSardone, Valentina
dc.contributor.authorAnwar, Rayan
dc.contributor.authorEl Andaloussi, Samir
dc.contributor.authorLehto, Taavi
dc.contributor.authorKhoo, Bernard
dc.contributor.authorBrolin, Camilla
dc.contributor.authorvan Roon-Mom, Willeke Mc
dc.contributor.authorGoyenvalle, Aurélie
dc.contributor.authorAartsma-Rus, Annemieke
dc.contributor.authorArechavala-Gomeza, Virginia
dc.date.accessioned2019-08-01T06:00:27Z-
dc.date.available2019-08-01T06:00:27Z-
dc.date.issued2017
dc.identifier.citationEMBO Mol Med.2017 05;(9)5:545-557
dc.identifier.urihttps://hdl.handle.net/20.500.12530/41247-
dc.description.abstractThe 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.
dc.language.isoeng
dc.rightsopenAccess-
dc.subjectRNA therapy
dc.subjectantisense oligonucleotides
dc.subjectdelivery
dc.subjectpre‐clinical models
dc.subjecttoxicity
dc.subject.meshAnimals
dc.subject.meshDrug Administration Routes
dc.subject.meshDrug Delivery Systems
dc.subject.meshDrug Evaluation, Preclinical
dc.subject.meshGenetic Therapy
dc.subject.meshHumans
dc.subject.meshOligonucleotides, Antisense
dc.subject.meshRNA Splicing
dc.titleDelivery is key: lessons learnt from developing splice-switching antisense therapies.
dc.typeArtículo
dc.identifier.pubmedID28289078
dc.format.volume9
dc.format.page545-557
dc.identifier.e-issn1757-4684
dc.identifier.journalEMBO molecular medicine
dc.identifier.doi10.15252/emmm.201607199
dc.format.number5
dc.identifier.pmcPMC5412803
dc.pubmedtypeJournal Article
dc.pubmedtypeReview
dc.pubmedtypeResearch Support, Non-U.S. Gov't
Appears in Collections:Fundaciones e Institutos de Investigación > IIS H. U. La Paz > Artículos

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