Overview of siRNA
Small interfering RNAs (siRNAs) are short double-strand RNA molecues less than 30 nt long. Once in the cells, they are loaded to enzyme complexes, which can recognize complimentary mRNA sequences and degrade them to achieve therapeutic effects.
siRNA drugs can be developed based on platform technologies. Their sequences are complementary to target mRNA according to base-pairing rules. Almost all genes can be down-regulated by siRNA, including traditionally undruggable targets. Combining with tissue-specific platform delivery technologies and chemical modifications, the potency, duration, and toxicity profile of siRNA drugs can be significantly improved. Thus, siRNA drugs can be developed a lot faster with much higher success rate. Super long-lasting efficacy and superb drug safety make them especially ideal for the treatment of chronic diseases and for certain rare diseases.
