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dc.contributor.author
Lightfoot, Helen L.
dc.contributor.author
Hall, Jonathan
dc.date.accessioned
2019-04-02T14:36:09Z
dc.date.available
2017-06-10T13:33:06Z
dc.date.available
2019-04-02T14:36:09Z
dc.date.issued
2012-11
dc.identifier.issn
1362-4962
dc.identifier.issn
0305-1048
dc.identifier.issn
1362-4954
dc.identifier.other
10.1093/nar/gks861
en_US
dc.identifier.uri
http://hdl.handle.net/20.500.11850/63006
dc.identifier.doi
10.3929/ethz-b-000063006
dc.description.abstract
Oligonucleotide delivery in vivo is commonly seen as the principal hurdle to the successful development of oligonucleotide drugs. In an analysis of 26 oligonucleotide drugs recently evaluated in late-stage clinical trials we found that to date at least half have demonstrated suppression of the target mRNA and/or protein levels in the relevant cell types in man, including those present in liver, muscle, bone marrow, lung, blood and solid tumors. Overall, this strongly implies that the drugs are being delivered to the appropriate disease tissues. Strikingly we also found that the majority of the drug targets of the oligonucleotides lie outside of the drugable genome and represent new mechanisms of action not previously investigated in a clinical setting. Despite the high risk of failure of novel mechanisms of action in the clinic, a subset of the targets has been validated by the drugs. While not wishing to downplay the technical challenges of oligonucleotide delivery in vivo , here we demonstrate that target selection and validation are of equal importance for the success of this field.
en_US
dc.format
application/pdf
en_US
dc.language.iso
en
en_US
dc.publisher
Oxford University Press
en_US
dc.rights.uri
http://creativecommons.org/licenses/by/3.0/
dc.title
Target mRNA inhibition by oligonucleotide drugs in man
en_US
dc.type
Journal Article
dc.rights.license
Creative Commons Attribution 3.0 Unported
ethz.journal.title
Nucleic Acids Research
ethz.journal.volume
40
en_US
ethz.journal.issue
21
en_US
ethz.journal.abbreviated
Nucleic Acids Res.
ethz.pages.start
10585
en_US
ethz.pages.end
10595
en_US
ethz.version.deposit
publishedVersion
en_US
ethz.identifier.nebis
000038633
ethz.publication.place
Oxford
en_US
ethz.publication.status
published
en_US
ethz.leitzahl
ETH Zürich::00002 - ETH Zürich::00012 - Lehre und Forschung::00007 - Departemente::02020 - Dep. Chemie und Angewandte Biowiss. / Dep. of Chemistry and Applied Biosc.::02534 - Institut für Pharmazeutische Wiss. / Institute of Pharmaceutical Sciences::03760 - Hall, Jonathan / Hall, Jonathan
en_US
ethz.leitzahl.certified
ETH Zürich::00002 - ETH Zürich::00012 - Lehre und Forschung::00007 - Departemente::02020 - Dep. Chemie und Angewandte Biowiss. / Dep. of Chemistry and Applied Biosc.::02534 - Institut für Pharmazeutische Wiss. / Institute of Pharmaceutical Sciences::03760 - Hall, Jonathan / Hall, Jonathan
ethz.date.deposited
2017-06-10T13:33:12Z
ethz.source
ECIT
ethz.identifier.importid
imp5936504e923f993334
ethz.ecitpid
pub:99927
ethz.eth
yes
en_US
ethz.availability
Open access
en_US
ethz.rosetta.installDate
2017-07-19T09:51:01Z
ethz.rosetta.lastUpdated
2019-04-02T14:36:29Z
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false
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true
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