Translational drug discovery approaches for fragile x-associated tremor/ataxia syndrome (FXTAS)

Abstract

Ribonucleic acid (RNA) is a multifunctional polymer that lies at the heart of any biological system. The biomolecule is typically single-stranded, which enables the formation of versatile 3D conformations that enable a diverse array of functions. A fundamental concept regarding RNA is that it serves as the intermediate biomolecule while transferring genetic information from DNA to proteins. Earlier, before the discovery of its catalytic property, RNA was recognized mainly for these three functions: a) acting as a messenger for DNA, b) serving as a bridge between amino acids and genetic codes, and c) forming a structural component of the ribosome. The discovery that RNA can also function as a catalyst has since revolutionized the field and opened new doors for scientists to explore the hidden aspects of cellular mechanisms. The effective functioning of a cell requires the precise and regulated expression of both protein-coding (mRNA) and non-coding RNA. In recent years, different types of RNA have been identified, each playing an indispensable role in governing cellular processes. RNAs are classified according to their structural features and contributions to cellular events.