eprintid: 1545307
rev_number: 36
eprint_status: archive
userid: 608
dir: disk0/01/54/53/07
datestamp: 2017-03-27 13:29:32
lastmod: 2021-12-10 01:13:06
status_changed: 2017-03-27 13:29:32
type: article
metadata_visibility: show
creators_name: Godfrey, C
creators_name: Desviat, LR
creators_name: Smedsrød, B
creators_name: Piétri-Rouxel, F
creators_name: Denti, MA
creators_name: Disterer, P
creators_name: Lorain, S
creators_name: Nogales-Gadea, G
creators_name: Sardone, V
creators_name: Anwar, R
creators_name: El Andaloussi, S
creators_name: Lehto, T
creators_name: Khoo, B
creators_name: Brolin, C
creators_name: van Roon-Mom, WM
creators_name: Goyenvalle, A
creators_name: Aartsma-Rus, A
creators_name: Arechavala-Gomeza, V
title: Delivery is key: lessons learnt from developing splice-switching antisense therapies
ispublished: pub
divisions: UCL
divisions: B02
divisions: C10
divisions: D17
divisions: G93
divisions: D14
keywords: RNA therapy, antisense oligonucleotides, delivery, pre‐clinical models, toxicity
note: 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.
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.
date: 2017-03-01
date_type: published
official_url: http://dx.doi.org/10.15252/emmm.201607199
oa_status: green
full_text_type: pub
language: eng
primo: open
primo_central: open_green
verified: verified_manual
elements_id: 1214486
doi: 10.15252/emmm.201607199
pii: emmm.201607199
lyricists_name: Disterer, Petra
lyricists_name: Khoo, Bernard
lyricists_id: PDIST62
lyricists_id: BKHOO30
actors_name: Disterer, Petra
actors_id: PDIST62
actors_role: owner
full_text_status: public
publication: EMBO Molecular Medicine
volume: 9
number: 3
pagerange: 281-394
event_location: England
issn: 1757-4684
citation:        Godfrey, C;    Desviat, LR;    Smedsrød, B;    Piétri-Rouxel, F;    Denti, MA;    Disterer, P;    Lorain, S;                                             ... Arechavala-Gomeza, V; + view all <#>        Godfrey, C;  Desviat, LR;  Smedsrød, B;  Piétri-Rouxel, F;  Denti, MA;  Disterer, P;  Lorain, S;  Nogales-Gadea, G;  Sardone, V;  Anwar, R;  El Andaloussi, S;  Lehto, T;  Khoo, B;  Brolin, C;  van Roon-Mom, WM;  Goyenvalle, A;  Aartsma-Rus, A;  Arechavala-Gomeza, V;   - view fewer <#>    (2017)    Delivery is key: lessons learnt from developing splice-switching antisense therapies.                   EMBO Molecular Medicine , 9  (3)   pp. 281-394.    10.15252/emmm.201607199 <https://doi.org/10.15252/emmm.201607199>.       Green open access   
 
document_url: https://discovery.ucl.ac.uk/id/eprint/1545307/1/Godfrey_2017%20Delivery%20is%20key%20-%20lessons%20learnt%20from%20developing%20splice-switching%20antisense%20therapies.pdf