Delivery is key: lessons learnt from developing splice‐switching antisense therapies
Delivery is key: lessons learnt from developing splice‐switching antisense therapies
About this item
Full title
Author / Creator
Godfrey, Caroline , Desviat, Lourdes R , Smedsrød, Bård , Piétri‐Rouxel, France , Denti, Michela A , Disterer, Petra , Lorain, Stéphanie , Nogales‐Gadea, Gisela , Sardone, Valentina , Anwar, Rayan , EL Andaloussi, Samir , Lehto, Taavi , Khoo, Bernard , Brolin, Camilla , van Roon‐Mom, Willeke MC , Goyenvalle, Aurélie , Aartsma‐Rus, Annemieke and Arechavala‐Gomeza, Virginia
Publisher
London: Nature Publishing Group UK
Journal title
Language
English
Formats
Publication information
Publisher
London: Nature Publishing Group UK
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More information
Scope and Contents
Contents
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 oligonucleot...
Alternative Titles
Full title
Delivery is key: lessons learnt from developing splice‐switching antisense therapies
Authors, Artists and Contributors
Author / Creator
Desviat, Lourdes R
Smedsrød, Bård
Piétri‐Rouxel, France
Denti, Michela A
Disterer, Petra
Lorain, Stéphanie
Nogales‐Gadea, Gisela
Sardone, Valentina
Anwar, Rayan
EL Andaloussi, Samir
Lehto, Taavi
Khoo, Bernard
Brolin, Camilla
van Roon‐Mom, Willeke MC
Goyenvalle, Aurélie
Aartsma‐Rus, Annemieke
Arechavala‐Gomeza, Virginia
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_dc24c7db81c9430cadef6dc5077b7915
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_dc24c7db81c9430cadef6dc5077b7915
Other Identifiers
ISSN
1757-4676,1757-4684
E-ISSN
1757-4684
DOI
10.15252/emmm.201607199
