RNAi and small interfering RNAs in human disease therapeutic applications

Small interfering RNA; principles, applications and challenges--

Gene silencing using RNAi (RNA interference), has recently been used as a successful laboratory technique in determining the function and control of gene expression and provides a wide range of applications in molecular biology and gene therapy. RNAi is a method of suppressing gene expression. In this direction, a single-stranded RNA molecule of about 21–23 nucleotides, called siRNA (small inte...

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...

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...

RNA interference and small interfering RNAs.

The term aRNA interferenceo (RNAi) was coined after the groundbreaking discovery that injection of double-stranded RNA (dsRNA) into the nematode Caenorhabditis elegans leads to specific silencing of genes highly homologous in sequence to the delivered dsRNA. The RNAi phenotype is either identical to the genetic null mutant or resembles an allelic series of mutants. The dsRNA can also be deliver...

Structure - Functions Relations in Small Interfering RNAs