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siRNAs represent one of the most revolutionary discoveries in modern biotechnology. They date back to the experiments of Andrew Fire and Craig Mello in 1998, who first described the phenomenon of RNA interference in the nematode C. elegans nematode and were awarded the Nobel Prize in Medicine in 2006. In their research, they observed how certain RNA molecules could silence the expression of specific genes, This opened the door to a whole new field of genetic research. This is why the term “silent revolution” is applied when talking about siRNAs. applies when talking about siRNAs.
From laboratories to therapy
The initial discovery of siRNAs led to further research into how these molecules could be manipulated for therapeutic use. siRNAs act by binding to a specific messenger RNA (mRNA), triggering its degradation and thereby preventing the production of specific proteins. This ability to “switch off” selected genes has prompted their study in several diseases, especially those in which overexpression of certain genes plays a central role, such as in cancer or viral infections. However, turning siRNAs into effective therapeutic tools has been a long process. Early challenges included the stability of siRNA molecules in the body and specific delivery to target cells. To overcome these obstacles, researchers have developed innovative methods of encapsulation and chemical modification that improve the stability and ability of siRNAs to reach the right cells. 
The potential of siRNAs in disease research and treatment
siRNAs have shown enormous potential in gene therapy. One of their most promising applications is in the treatment of genetic diseases, where defects in a single gene can be corrected by silencing the defective messenger RNA. They have also been explored in cancer treatment, by reducing the expression of specific oncogenes, and in viral diseases such as hepatitis B and HIV, where they inhibit viral replication by destroying viral messenger RNAs. Since 2018, the FDA has approved six siRNA-based therapies: Patisiran, Givosiran, Lumasiran, Inclisiran, Nedosiran and Vutisiran. The role of siRNA molecules in precision medicine is crucial, as they enable a highly targeted approach to disease treatment. Increasingly, medicine is moving towards personalised treatments, where the underlying genetic cause of a disease is addressed. In this context, siRNAs are powerful tools, as they can be designed to specifically target individual genetic mutations, opening up a new era of targeted therapies. siRNA molecules have evolved from their discovery as basic genetic research tools to become promising therapies for a wide range of diseases, including some of the most difficult to treat. At OLIGOFASTX, our partner Sylentis takes advantage of the latest advances in artificial intelligence during the design of siRNAs to avoid drug-related risks, such as toxicological risks, from the very beginning of their development.
Sources:
Molecular Mechanisms and Biological Functions of siRNA – PMC
How the RNAi therapeutic ‘operating system’ is rewriting drug discovery
Clinical Advances of siRNA-Based Nanotherapeutics for Cancer Treatment – PMC
Images:
Revolution in mRNA vaccine technology using AI | College of Engineering | Oregon State University
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