Therapeutic potential of chemically modified si RNA : Recent trends
Small interfering RNA s (si RNA s) are one of the valuable tools to investigate the functions of genes and are also used for gene silencing. It has a wide scope in drug discovery through in vivo target validation. si RNA therapeutics are not optimal drug‐like molecules due to poor bioavailability an...
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Veröffentlicht in: | Chemical biology & drug design 2017-11, Vol.90 (5), p.665-678 |
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Hauptverfasser: | , , , , |
Format: | Artikel |
Sprache: | eng |
Online-Zugang: | Volltext |
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Zusammenfassung: | Small interfering
RNA
s (si
RNA
s) are one of the valuable tools to investigate the functions of genes and are also used for gene silencing. It has a wide scope in drug discovery through in vivo target validation. si
RNA
therapeutics are not optimal drug‐like molecules due to poor bioavailability and immunogenic and off‐target effects. To overcome the challenges associated with si
RNA
therapeutics, identification of appropriate chemical modifications that improves the stability, specificity and potency of si
RNA
is essential. This review focuses on the various chemical modifications and their implications in si
RNA
therapy. |
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ISSN: | 1747-0277 1747-0285 |
DOI: | 10.1111/cbdd.12993 |