Development of Therapeutic-Grade Small Interfering RNAs by Chemical Engineering

Development of Therapeutic-Grade Small Interfering RNAs by Chemical Engineering

Recent successes in clinical trials have provided important proof of concept that small interfering RNAs (siRNAs) indeed constitute a new promising class of therapeutics. Although great efforts are still needed to ensure efficient means of delivery in vivo, the siRNA molecule itself has been successfully engineered by chemical modification to meet initial challenges regarding specificity, stabi...

Non-Viral Delivery and Therapeutic Application of Small Interfering RNAs

RNA interference (RNAi) is a powerful method used for gene expression regulation. The increasing knowledge about the molecular mechanism of this phenomenon creates new avenues for the application of the RNAi technology in the treatment of various human diseases. However, delivery of RNA interference mediators, small interfering RNAs (siRNAs), to target cells is a major hurdle. Effective and saf...

Rotavirus gene silencing by small interfering RNAs.

RNA interference is an evolutionarily conserved double-stranded RNA-triggered mechanism for suppressing gene expression. Rotaviruses, the leading cause of severe diarrhea in young children, are formed by three concentric layers of protein, from which the spike protein VP4 projects. Here, we show that a small interfering RNA corresponding to the VP4 gene efficiently inhibits the synthesis of thi...

Design of active small interfering RNAs.

Small interfering RNAs (siRNAs) have become the experimental tool of choice to suppress gene expression in a wide variety of organisms. Site selection and optimization does not appear to be as difficult for siRNAs as would be expected from historical experience with antisense oligonucleotides. Nevertheless, not all sites within a target gene perform equally. Significant progress has been made i...

stably silencing of cd81 expression by small interfering rnas targeting 3'-ntr inhibits hcv infection