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Recent Update on siRNA Therapeutics

Oluwakemi Ebenezer, Abel Kolawole Oyebamiji, John Oludele Olanlokun, Jack A. Tuszyński, Gane Ka‐Shu Wong

2025International Journal of Molecular Sciences34 citationsDOIOpen Access PDF

Abstract

Small interfering RNA (siRNA) has been deemed a promising therapeutic method for treating diverse diseases. siRNA-based therapeutics provide a distinct mechanism of action by selectively targeting and silencing disease-causing genes at the post-transcriptional level. This paper provides an overview of the present state of siRNA-based therapeutics, highlighting their potential in different therapeutic areas. The first section of this review introduces the basic principles of siRNA technology, including its mechanism of action and delivery methods. Subsequently, we discuss the impediments associated with siRNA delivery and manufacturing development and the strategies for overcoming these obstacles. The clinical advancement of siRNA therapeutics in various disease areas, including cancer, genetic disorders, viral infections, and inflammatory diseases, is summarized. Lastly, we summarize the successes, failures, and lessons learned from the development of siRNAs. With advancements in delivery systems and improvements in target selection, the field of medicine can be revolutionized, and siRNA therapeutics can offer new treatment options for patients.

Topics & Concepts

Small interfering RNAGene silencingMechanism (biology)Computational biologyDiseaseRNA interferenceMedicineBioinformaticsBiologyRNAGeneGeneticsPathologyEpistemologyPhilosophyRNA Interference and Gene DeliveryAdvanced biosensing and bioanalysis techniquesMicroRNA in disease regulation