Animal RNAi studies are experimental studies where RNA interference (RNAi) is used to specifically target and knockdown the expression of genes in animal models. RNAi is a powerful tool for studying gene function, as it allows for the rapid and specific inhibition of gene expression in cells and organisms.
Animal RNAi studies typically involve the delivery of small interfering RNAs (siRNAs) or short hairpin RNAs (shRNAs) to the animal model of interest, either by injection or through genetic modification of the animal’s cells. Once delivered, the siRNAs or shRNAs can specifically target and degrade messenger RNAs (mRNAs) encoding the gene of interest, leading to decreased expression of the protein and allowing researchers to study the effects of this knockdown on the animal’s phenotype.
Animal RNAi studies have been used to study a wide range of biological processes and diseases, including cancer, neurodegenerative diseases, and developmental biology. These studies have provided valuable insights into the function of specific genes and pathways, and have identified potential targets for therapeutic intervention.
In addition to providing insights into gene function and disease biology, animal RNAi studies have also been used to develop RNA-based therapies for human diseases. For example, the first RNAi-based drug, Onpattro, was approved by the US Food and Drug Administration in 2018 for the treatment of hereditary transthyretin-mediated amyloidosis.
Overall, animal RNAi studies are a powerful tool for studying gene function, disease biology, and developing new therapies. They allow researchers to specifically target and knockdown the expression of genes in animal models, providing valuable insights into the function of specific genes and pathways.