RNA Interference: A Potential Approach For Silencing Splice Isoforms Associated With Human Diseases
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Abstract
Double-stranded RNA (dsRNA) initiates the post-transcriptional gene silencing process known as RNA interference. Small interfering RNA (siRNA), short hairpin RNA (shRNA), and bi-functional shRNA are techniques for mediating the RNAi effect. For some medical conditions (such viral injections), the ease of generating siRNA and the brief duration of each dosage make them ideal candidates. Optimized shRNA constructs, particularly those contained in a miRNA scaffold, provide high potency and long-lasting effects with low copy numbers and fewer off-target effects by leveraging the endogenous processing machinery. RNAi-based therapies' efficacy and safety might be further improved by bi-functional design. There is a great deal of interest in employing RNAi as a technique in many different contexts because of its specificity and resilience. Recent developments in the use of RNAi, a method for regulating normal gene expression, as a possible therapeutic agent for a variety of illnesses and disorders, including cancer, infectious diseases, and metabolic disorders. Clinical trials for RNAi have started, but significant challenges still need to be addressed before RNAi can be thought of as a conventional medicine, including off-target effects, toxicity, and dangerous delivery systems. In this article, we initially examined the RNAi mechanism before concentrating on some of its biological research uses, including the therapy of HIV, viral hepatitis, and a number of other disorders.
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