Fundamentally, RNAi is a natural process of post-transcriptional gene silencing, involving short strands of nucleic acids. Cells use this process to silence and / or inhibit gene expression, via the targeted degradation of specific (unwanted) mRNA molecules. From an application perspective, the gene specificity of RNAi is the primary reason why it is being considered for therapy development. In theory, RNAi based therapeutics are capable of treating indications, such as age-related macular degeneration (AMD), hepatitis C and various forms of cancer, which are actually hard to treat, using conventional pharmacological options.
Since 2011, there has been an increase in the number of candidate therapies in the clinical pipeline as companies are developing novel delivery methods and anti-immuno-stimulatory strategies based on sound scientific rationale. The current clinical dataflow is set to be a major attractor for rich biotech schemes as RNAi is the next hot area in drug development.
RNAi technology has emerged as a powerful tool to analyze various genes in a variety of organisms. This technology can be used in functional genomics as it is highly specific and can silence a particular gene from a multiple gene family. Since, RNAi depends on sequence homology, it enables the selection of unique / conserved regions of target genes for silencing.
RNAi technology offers the promise of being able to treat diseases that are characterized by abnormal gene functions. It targets a disease at post-transcriptional level and thus, is a highly selective technology. The most common therapeutic areas that have captured the focus of both research institutions and companies are neurogenerative disorders, oncology and viral diseases.
It is worth mentioning that, Onpattro, a RNAi based therapeutic drug, recently received marketing approval for the treatment of Hereditary ATTR Amyloidosis. Currently, there are many clinical trials going on, to study the safety and efficacy of siRNAs molecules for the treatment of various infectious diseases. Viral diseases that can be potentially treated using RNAi include hepatitis B, hepatitis C, HIV and influenza. In addition, RNAi technology has the potential to transform the treatment regimen for various cancers. Due to its reduced cytotoxic effects, this technology is regarded safe. Further, it is highly specific and only involves the knockdown of its target gene.
Further, genome wide screening can be achieved through gene knockdowns or by using RNAi for the identification of genes that influence a particular phenotype. The use of RNAi in genome screening provides a vast amount of data per experiment as it interacts with thousands of genes. The basic process of genome wide screening experiments involves the use of a RNAi library, stable cell types, transfection with RNAi agents, signal detection and analysis and identification of genes for therapeutic purposes.
With two approved drug and several therapy candidates in late stages of clinical development, the field of RNAi therapeutics presents significant opportunity for interested biopharmaceutical developers. This drug class has the potential to treat a variety of clinical conditions, including oncological disorders, genetic diseases, hepatic diseases, respiratory disorders and infectious diseases.
One of the major challenges in this domain is related to the delivery of RNAi therapeutics. However, in recent years, several types of novel delivery systems have been developed and are being investigated for therapeutic nucleic acid delivery. The key aim in this context is to increase the efficiency of drug delivery to the target site. Examples of the various delivery systems, which are either already being used, or under evaluation, include lipid nanoparticle delivery, polypeptide delivery and conjugated delivery system. In summary, RNAi-based therapeutics are expected to soon become one of the prominent therapeutic options within mainstream healthcare.
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 Source: https://www.intechopen.com/books/functional-genomics/rnai-towards-functional-genomics-studies
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