Project: RNAi Therapeutics: The Incredible Potential of siRNAs

This mechanism-of-action animation highlights the therapeutic potential of small interfering RNAs (siRNAs), a powerful approach within RNA interference (RNAi), including an innovative siRNA delivery platform known as Targeted RNAi Molecules.

Above: A view of siRNAs

Many diseases stem from the overproduction of specific proteins or the presence of mutant proteins. Since proteins are synthesized from their corresponding mRNA, RNAi utilizes a natural biological process to selectively “silence” specific mRNAs. This reduces the production of disease-causing proteins and offers a targeted and precise approach to treatment.

The animation begins by illustrating the normal process of protein synthesis before exploring the mechanism of siRNA delivery to cells. It also illustrates how siRNAs guide RNAi machinery to degrade specific mRNAs, effectively silencing target genes and reducing the production of harmful proteins. Additionally, it introduces a promising treatment designed to overcome three critical challenges: achieving targeted delivery, ensuring sustained duration, and preventing siRNA drugs from affecting off-target genes.