An overview of the clinical application of antisense oligonucleotides for RNA-targeting therapies
Highlights • Recent human clinical trial data on RNAi approaches to gene down-regulation for genetic diseases. • Overview of current anti-microRNA drugs with specific emphasis on application to target Hepatitis C infection. • Update on the current state of ‘exon skipping’ field for both Duchenne mus...
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Veröffentlicht in: | Current opinion in pharmacology 2015-10, Vol.24, p.52-58 |
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Hauptverfasser: | , |
Format: | Artikel |
Sprache: | eng |
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Online-Zugang: | Volltext |
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Zusammenfassung: | Highlights • Recent human clinical trial data on RNAi approaches to gene down-regulation for genetic diseases. • Overview of current anti-microRNA drugs with specific emphasis on application to target Hepatitis C infection. • Update on the current state of ‘exon skipping’ field for both Duchenne muscular dystrophy and spinal muscular atrophy. |
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ISSN: | 1471-4892 1471-4973 |
DOI: | 10.1016/j.coph.2015.07.005 |