RNA therapeutics: Identification of novel targets leading to drug discovery.

Abstract

The established concept that RNA works only for protein synthesis has been changed over the past few decades and shifted towards therapeutic purposes. Almost 98% of mammalian genome is transcribed into nonprotein coding RNA termed as noncoding RNA (ncRNA) which plays regulatory role in molecular and cellular functions as controlling gene expression. These ncRNAs are classified as long noncoding RNA (lncRNA), short noncoding RNA (sncRNA), and translational/structural RNA which possess diverse functions. These ncRNAs regulate expression of normal gene and modulate disease development and progression. The characterization of ncRNA genes and their mechanisms can aid in disease diagnosis, examining its development and direct specific therapies in different disease treatments. Due to their unique modes of action, they are designated as novel class of targets leading to drug discovery. The modulation in these ncRNAs can enhance therapeutic treatments against different diseases by targeting mRNA for its cleavage via antisense olionucleotides (ASOs)/DNA duplex, RNA alternative splicing/editing, chromatin modification, transcriptional/translational interference, RNA masking, small interfering RNA/microRNA-based gene silencing and by inducing immunity via RNA-based vaccination. Here in this review, we tried to summarize the emerging fields of ncRNA, their role in different diseases, their modes of action, and their potential in target identification and therapeutic drug development.