SIRNA: ITS TRANSLATIONFROM RESEARCH TO THERAPEUTIC APPLICATIONS

Small interfering RNA (siRNA) is a short, double-stranded RNA molecule that has emerged as a pivotal tool for gene silencing through the RNA interference (RNAi) pathway. It is a powerful post-transcriptional gene-silencing molecule central to RNA interference (RNAi) mechanisms1. Typically 21–25 nucleotides in length, it guides the RNA-induced silencing complex (RISC) to a target messenger RNA (mRNA), enabling sequence-specific degradation and effectively silencing gene expression2. This process is highly specific and forms the foundation for siRNA’s role in research and therapeutic interventions3. By guiding the RNA-induced silencing complex (RISC) to target mRNA, siRNA enables post-transcriptional gene regulation with unparalleled precision. This targeted action holds immense promise in treating genetic, infectious, and degenerative diseases4. Therapeutically, siRNA offers unique advantages over traditional treatments, including the ability to target undruggable proteins, rapid development timelines, and reduced systemic toxicity. These advantages have catalyzed interest in siRNA for precision medicine and rare genetic disorders5