Recent trends and spotlight on nucleotide-based drugs: novel targets, their design, delivery, and clinical potential

Abstract

Therapeutics comprising of nucleotide entities is very broad class, covers nucleotide analogs, oligonucleotides and nucleic acid-based based therapeutic. Nucleotide/nucleoside-based drugs are already very well explored, with clinically approved candidates as antiviral, anti-cancer, anti-bacterial and anti-rheumatoid etc (Garner, 2021). Oligonucleotide therapy is relative recent and promising, comprising of Antisense Oligonucleotides (ASOs), Small Interfering RNAs (siRNAs), Short Hairpin RNAs (shRNAs), Anti-MicroRNAs (anti-miRs). Oligonucleotides selectively bind to RNA or proteins, blocking their function or promoting degradation. Oligonucleotide therapy shows potential in treating genetic disorders, cancer, viral infections, and neurodegenerative conditions