Delivery is Key: Lessons Learnt from Developing Splice-switching Antisense Therapies

Overview

Journal EMBO Mol Med

Specialty Molecular Biology

Date 2017 Mar 15

PMID 28289078

Citations 78

Authors

Caroline Godfrey

Lourdes R Desviat

Bard Smedsrod

France Pietri-Rouxel

Michela A Denti

Petra Disterer

Stephanie Lorain

Gisela Nogales-Gadea

Valentina Sardone

Rayan Anwar

Samir El Andaloussi

Taavi Lehto

Bernard Khoo

Camilla Brolin

Willeke Mc van Roon-Mom

Aurelie Goyenvalle

Annemieke Aartsma-Rus

Virginia Arechavala-Gomeza

Affiliations

Soon will be listed here.

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.

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