The discovery of RNA interference (RNAi) was first made in petunia plants. Scientists Andrew Fire and Craig Mellow were awarded the Nobel Prize in Physiology or Medicine in 2006 for their discovery of RNA interference. They conducted their research using the nematode worm Caenorhabditis elegans as a model organism.
RNA interference pathways are naturally present in various eukaryotic cells and silence gene expression at the post-transcriptional level, thereby controlling gene expression. In these pathways, small ribonucleic acid molecules, specifically small interfering RNA (siRNA) and micro-RNA (miRNA), play a crucial role.
In the RNAi pathway, the initial step involves processing double-stranded RNA or primary micro-RNA transcripts into siRNA by the RNase III enzyme Dicer. The next step is the loading of siRNA into the RNA-induced silencing complex (RISC). During RISC assembly, siRNA unwinds, and the single-stranded RNA hybridizes with messenger RNA (mRNA). The targeted mRNA is then degraded by the RNase H enzyme, resulting in gene silencing. Mismatches in the siRNA/mRNA duplex will not lead to cleavage.
The discovery of RNA interference has paved the way for extensive research and applications in various fields, including medicine, cancer treatment, dominant genetic disorders, viral infections, understanding neurodegenerative disorders, the food industry, and the development of transgenic plants, among others.
Due to its significant characteristics, RNAi is a valuable tool in gene therapy studies and holds potential as a treatment for various genetic disorders.
References
Weiss B, Davidkova G, Zhou LW (March 1999). "Antisense RNA gene therapy for studying and modulating biological processes". Cellular and Molecular Life Sciences. 55 (3): 334–58.
Lee RC, Feinbaum RL, Ambros V (December 1993). "The C. elegans heterochronic gene lin-4 encodes small RNAs with antisense complementarity to lin-14". Cell. 75 (5): 843–54.
Wall NR, Shi Y (October 2003). "Small RNA: can RNA interference be exploited for therapy?". Lancet. 362 (9393): 1401–3.
Sah D (2006). "Therapeutic potential of RNA interference for neurological disorders". Life Sci. 79 (19): 1773–80.
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