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

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

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 approac...

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Main Authors: Caroline Godfrey, Lourdes R Desviat, Bård Smedsrød, France Piétri‐Rouxel, Michela A Denti, Petra Disterer, Stéphanie Lorain, Gisela Nogales‐Gadea, Valentina Sardone, Rayan Anwar, Samir EL Andaloussi, Taavi Lehto, Bernard Khoo, Camilla Brolin, Willeke MC van Roon‐Mom, Aurélie Goyenvalle, Annemieke Aartsma‐Rus, Virginia Arechavala‐Gomeza
Format: Article
Language:English
Published: Springer Nature 2017-03-01
Series:EMBO Molecular Medicine
Subjects:
antisense oligonucleotides
delivery
pre‐clinical models
RNA therapy
toxicity
Online Access:https://doi.org/10.15252/emmm.201607199
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https://doi.org/10.15252/emmm.201607199

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