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Antisense-mediated splice intervention to treat human disease: the odyssey continues

Recent approvals of oligonucleotide analogue drugs to alter gene expression have been welcomed by patient communities but not universally supported. These compounds represent a class of drugs that are designed to target a specific gene transcript, and they include a number of chemical entities to ev...

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Bibliografische gegevens
Gepubliceerd in:F1000Res
Hoofdauteurs: Pitout, Ianthe, Flynn, Loren L., Wilton, Steve D., Fletcher, Sue
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: F1000 Research Limited 2019
Onderwerpen:
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6534073/
https://ncbi.nlm.nih.gov/pubmed/31164976
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.12688/f1000research.18466.1
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