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Abstract

SMALL INTERFERING RNA (SIRNA) OPERATES BY BINDING TO SPECIFIC MRNA SEQUENCES, WHICH LEADS TO THE DEGRADATION OF THE MRNA MOLECULES. THIS MECHANISM MAKES SIRNA A PROMISING THERAPEUTIC STRATEGY FOR TREATING VARIOUS DISEASES THAT STEM FROM ABNORMAL GENE EXPR

*Utkarsh R. Mandage, Jagruti R. Patil, Indrayani S. Pagare, Dr. Sarita M. Beldar, Shreya Bhausaheb Shirsath

ABSTRACT

Small interfering RNA (siRNA) represents a revolutionary approach in molecular biology, serving as a powerful tool for the regulation of gene expression. siRNA operates by binding to specific messenger RNA (mRNA) sequences, initiating the RNA interference (RNAi) pathway, which leads to the degradation of the target mRNA molecules. This RNAi mechanism has positioned siRNA as a promising therapeutic strategy for treating a variety of diseases caused by abnormal or dysregulated gene expression, such as genetic disorders, viral infections, and cancers. The ability of siRNA to precisely silence genes implicated in disease progression has attracted significant attention in the field of therapeutic development. However, challenges related to the delivery, stability, and specificity of siRNA remain as key obstacles that must be overcome to ensure its successful clinical application. This paper provides an in-depth analysis of the mechanism by which siRNA mediates gene silencing, highlights its potential for treating diseases associated with aberrant gene expression, and discusses the current strategies being employed to enhance the effectiveness of siRNA therapies. Furthermore, the paper explores the ongoing research and clinical trials that have showcased the therapeutic potential of siRNA in treating diseases like cancer, viral infections, and genetic disorders. In addition, we examine the innovative techniques being developed to improve the stability and targeted delivery of siRNA, which are crucial for its future success in clinical settings. By addressing these challenges, siRNA could emerge as a transformative therapeutic modality, offering hope for patients suffering from conditions that have previously been difficult to treat.

Keywords: Small interfering RNA (siRNA), gene silencing, RNA interference (RNAi), mRNA degradation, therapeutic strategy, gene expression regulation, genetic disorders, cancer, viral infections, therapeutic development, delivery systems, targeted therapy, molecular


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