Abstract

THERAPEUTIC APPLICATIONS OF OLIGONUCLEOTIDES

Aishwarya S. Shinde*, Asmita A. Tigote and Prajakta D. Nayakal

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Abstract

RNA is a unique therapeutics development target. Some of the advantages in using RNA as therapeutic target include applicability up to most RNAs in the cell, including noncoding RNAs, translation of genetic findings directly into drug discovery programs, and speeding and efficiency of the drug discovery process. While some promising advances have been made in the identification of small-molecule drugs that can modulate RNA function, oligonucleotides offer a much more direct—and, therefore, more expeditious—route. Antisense oligonucleotides are a new therapeutic platform for drug discovery with enormous potential to treat the multitude of diseases. Now, oligonucleotides stand to serve large populations of patients. Until now, their action used to be against diseases found in only a small number of patients. That being said, improvements can still be made using the oligonucleotide technology, with probably more discoveries set to be found in the coming years. Examples of the oligonucleotide therapeutics include antisense, oligonucleotides, small interfering RNA, microRNAs, aptamers, and decoys. The heterogeneity and feasibility of these diagnostically or therapeutically active drugs have increased incredibly over the past 25 years. There has been an emergence of several clinical and preclinical studies of oligonucleotides in patients with various neurodegenerative diseases, infectious diseases, respiratory diseases and in various types of cancer. This paper presents a summary of basic information, mechanism of action and applications of antisense oligonucleotides, small interfering RNA, microRNAs, aptamers and decoys.

Keywords: Antisense oligonucleotides, siRNAs, miRNAs, Aptamers, Decoys, Ribonucleic acid.