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 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.
Department of Molecular and Cellular Biology and Irell and Manella Graduate School of Biological Sciences, Beckman Research Institute at City of Hope, Duarte, California 91010, USA.
Nicholas M
NM
Snead
John J
JJ
Rossi
D002986:Clinical Trials as Topic; D006801:Humans; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000008:administration & dosage / Q000627:therapeutic use*
D034741:RNA, Small Interfering