Small interfering RNA: Discovery, pharmacology and clinical development—An introductory review

Post‐transcriptional gene silencing targets and degrades mRNA transcripts, silencing the expression of specific genes. RNA interference technology, using synthetic structurally well‐defined short double‐stranded RNA (small interfering RNA [siRNA]), has advanced rapidly in recent years. This introduc...

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Veröffentlicht in:	British journal of pharmacology 2023-11, Vol.180 (21), p.2697-2720
Hauptverfasser:	Ranasinghe, Priyanga, Addison, Melisande L., Dear, James W., Webb, David J.
Format:	Artikel
Sprache:	eng
Schlagworte:	Amyloidosis Bioavailability Chemical modification Gene expression Gene silencing Genetic disorders Hypercholesterolemia Hyperoxaluria Immune response Liver Nuclease Patient compliance Pharmacodynamics Pharmacokinetics Porphyria Primary hyperoxaluria RNA-mediated interference siRNA Transthyretin
Online-Zugang:	Volltext
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