RNA Interference Trigger Variants: Getting the Most Out of RNA for RNA Interference-Based Therapeutics

The manifestation of RNA interference (RNAi)-based therapeutics lies in safe and successful delivery of small interfering RNAs (siRNAs), the molecular entity that triggers and guides sequence-specific degradation of target mRNAs. Optimizing the chemistry and structure of siRNAs to achieve maximum ef...

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Veröffentlicht in:Nucleic acid therapeutics 2012-06, Vol.22 (3), p.139-146
Hauptverfasser: Snead, Nicholas M., Rossi, John J.
Format: Artikel
Sprache:eng
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Zusammenfassung:The manifestation of RNA interference (RNAi)-based therapeutics lies in safe and successful delivery of small interfering RNAs (siRNAs), the molecular entity that triggers and guides sequence-specific degradation of target mRNAs. Optimizing the chemistry and structure of siRNAs to achieve maximum efficacy is an important parameter in the development of siRNA therapeutics. The RNAi protein machinery can tolerate a variety of non-canonical modifications made to siRNAs, each of which imparts advantageous properties. Here, we review these modifications to siRNAs in pre-clinical and clinical studies.
ISSN:2159-3337
2159-3345
DOI:10.1089/nat.2012.0361