Molecular Mechanisms and Biological Functions of siRNA

TLDR

siRNA regulates gene expression through RNA interference, a discovery that revolutionized biomedical research and positioned siRNA as a versatile therapeutic and investigative tool for targeting genes in genetic diseases, cancer, and other conditions. The study aims to develop carriers that effectively deliver siRNA to its intended site of action for therapeutic use. siRNA delivery systems are divided into viral and non‑viral types, with non‑viral carriers such as polymers, lipids, and peptides forming supramolecular complexes that facilitate targeted delivery.

Abstract

One of the most important advances in biology has been the discovery that siRNA (small interfering RNA) is able to regulate the expression of genes, by a phenomenon known as RNAi (RNA interference). The discovery of RNAi, first in plants and Caenorhabditis elegans and later in mammalian cells, led to the emergence of a transformative view in biomedical research. siRNA has gained attention as a potential therapeutic reagent due to its ability to inhibit specific genes in many genetic diseases. siRNAs can be used as tools to study single gene function both in vivo and in-vitro and are an attractive new class of therapeutics, especially against undruggable targets for the treatment of cancer and other diseases. The siRNA delivery systems are categorized as non-viral and viral delivery systems. The non-viral delivery system includes polymers; Lipids; peptides etc. are the widely studied delivery systems for siRNA. Effective pharmacological use of siRNA requires 'carriers' that can deliver the siRNA to its intended site of action. The carriers assemble the siRNA into supramolecular complexes that display functional properties during the delivery process.