Open AccessDelivery is key: lessons learnt from developing splice‐switching antisense therapies
Author(s) -
Godfrey Caroline,
Desviat Lourdes R,
Smedsrød Bård,
PiétriRouxel France,
Denti Michela A,
Disterer Petra,
Lorain Stéphanie,
NogalesGadea Gisela,
Sardone Valentina,
Anwar Rayan,
EL Andaloussi Samir,
Lehto Taavi,
Khoo Bernard,
Brolin Camilla,
RoonMom Willeke MC,
Goyenvalle Aurélie,
AartsmaRus Annemieke,
ArechavalaGomeza Virginia
Publication year - 2017
Publication title -
embo molecular medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 4.923
H-Index - 107
eISSN - 1757-4684
pISSN - 1757-4676
DOI - 10.15252/emmm.201607199
Subject(s) - clinical trial , medicine , oligonucleotide , translation (biology) , bioinformatics , computational biology , computer science , biology , genetics , messenger rna , gene , dna
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.