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

Godfrey, C; Desviat, LR; Smedsrød, B; Piétri-Rouxel, F; Denti, MA; Disterer, P; Lorain, S; ... Arechavala-Gomeza, V; + view all (2017) Delivery is key: lessons learnt from developing splice-switching antisense therapies. EMBO Molecular Medicine , 9 (3) pp. 281-394. 10.15252/emmm.201607199. Green open access

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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.

Type: Article
Title: Delivery is key: lessons learnt from developing splice-switching antisense therapies
Location: England
Open access status: An open access version is available from UCL Discovery
DOI: 10.15252/emmm.201607199
Publisher version: http://dx.doi.org/10.15252/emmm.201607199
Language: English
Additional information: This is an open access article under the terms of the Creative Commons Attribution 4.0 License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
Keywords: RNA therapy, antisense oligonucleotides, delivery, pre‐clinical models, toxicity
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine > Renal Medicine
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Institute of Cardiovascular Science
URI: https://discovery.ucl.ac.uk/id/eprint/1545307
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