Ribonucleic acid and RNA-associated processes (RNA processes)

RNA and RNA-associated processes encompass the synthesis, maturation, transport, and translation of ribonucleic acids, serving as the critical link between the genome and the proteome (NIH, 2023). This broad category includes various RNA species such as messenger RNA (mRNA), transfer RNA (tRNA), ribosomal RNA (rRNA), and non-coding RNAs like microRNAs and long non-coding RNAs (Warner et al., 2018). In many diseases, these processes are dysregulated; for instance, aberrant splicing leads to genetic disorders, while overexpressed oncogenic mRNAs drive cancer progression (Crooke et al., 2021). Historically, RNA was considered "undruggable," but recent advances in antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and small molecules that target RNA secondary structures have revolutionized the field (Zhu et al., 2022). Drugs like Nusinersen modulate splicing to treat spinal muscular atrophy, while mRNA vaccines represent a breakthrough in infectious disease prevention (FDA, 2023). Targeting RNA-associated processes allows for the modulation of proteins that are otherwise difficult to target directly, offering a versatile therapeutic platform across oncology, neurology, and rare genetic conditions.

Other names

RNA metabolismRNA processingTranscriptome regulationRibonucleic acid

Drugs targeting RNA and its processes typically function through antisense-mediated splicing modulation, RNA interference (RNAi) for gene silencing, direct inhibition of viral RNA-dependent RNA polymerases, or binding to bacterial ribosomal RNA to inhibit protein synthesis (Warner et al., 2018; Crooke et al., 2021).