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
Hauptverfasser: McClorey, Graham, Wood, Matthew J
Format: Artikel
Sprache:eng
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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.
ISSN:1471-4892
1471-4973
DOI:10.1016/j.coph.2015.07.005