RNA-Based Therapeutics
摘要
Ribonucleic acid (RNA) therapeutics is an advanced approach of using RNA molecules to regulate gene expression for targeting genetic and acquired diseases as they are highly effective in targeting, otherwise, undruggable sites in the human body. This chapter aims to explore the types of RNA therapeutics such as antisense oligonucleotides, small interfering RNAsSmall interfering RNAsRNA therapeuticssmall interfering RNAs, microRNAs, and aptamers along with an overview of Food and Drug Administration-approved RNA therapeutics being utilized for the treatment of various disorders. Various factors that can improve the efficacy of RNA therapeutics have also been discussed. These include RNA stability and translation, transfection and induced immunity, and chemical modifications of RNAs as these factors are crucial for maximizing therapeutic outcomes by reducing off-target effects while prioritizing effective treatment. The advantages and effective delivery systems used in RNA therapeutics including nanoparticles and polymeric carriers to present ways of delivering therapeutic agents with minimal degradation for improved results have also been discussed. Additionally, it provides an update on RNA therapeutics undergoing clinical trials. At the end of this chapter, limitations in RNA therapeutics along with future directions are discussed for further research in utilizing these innovative techniques for the advancement in the field. This chapter, hence, provides the essential knowledge of advances in RNA therapeutics to improve treatment outcomes while limiting the adverse effects.