RNA silencing and applications

What is RNA interference?

RNA interference (RNAi) is a natural cellular mechanism that occurs in plants, animals and humans. It is mediated by small double-stranded RNA fragments called siRNAs that play a key role in gene regulation during development and in the immune response to viral infections.

Thanks to biotechnology, it is possible to take advantage of this cellular process to design targeted therapies based on RNAi. 

Some diseases are caused by a malfunction of proteins or by an excessive production of proteins. The use of drugs based on RNAi makes it possible to reduce or specifically control the production of those involved in a pathology.

In 2006 the Nobel Prize in Physiology or Medicine was awarded jointly to Andrew Z. Fire and Craig C. Mello for their discovery of siRNA-mediated RNAi. Twelve years later the first drug based on this technology was approved and made available to patients.

What is RNAi silencing?

Small interfering RNA (siRNA) molecules block, with high specificity, gene expression, the process by which information from a gene is used in the synthesis of a functional protein. 

RNAi drugs use the cellular machinery in the cytoplasm to silence messenger RNA (mRNA), which are molecular precursors of proteins, and thus, without affecting the genes, regulate their production in order to obtain a benefit for the treatment of the disease.