Single-stranded ribonucleic acid (ssRNA) (ssRNA)

Single-stranded ribonucleic acid (ssRNA) is a fundamental biological molecule that serves as the intermediary template (mRNA) for protein synthesis and a critical regulator of gene expression through non-coding forms such as microRNA and long non-coding RNA [1]. In the context of infectious diseases, ssRNA constitutes the genetic material for a wide array of human pathogens, including SARS-CoV-2, influenza, and Ebola, making the replication and stability of these viral genomes primary targets for antiviral drugs [2]. Therapeutically, ssRNA is targeted using sequence-specific technologies like antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs), which can induce the degradation of disease-causing transcripts or alter splicing patterns to treat genetic conditions such as spinal muscular atrophy [3]. Furthermore, exogenous or misplaced endogenous ssRNA acts as a pathogen-associated molecular pattern (PAMP) that is recognized by the innate immune system via endosomal Toll-like receptors 7 and 8 (TLR7/8), triggering the production of type I interferons and pro-inflammatory cytokines [4]. While RNA-targeted therapies offer high precision, significant challenges include the efficient delivery of these molecules to target tissues and the management of potential off-target effects or unintended immune stimulation [5]. [1] Alberts B, et al. (2014). Molecular Biology of the Cell. [2] NIH. Viral Genomes. [3] Crooke ST, et al. (2018). RNA-Targeted Therapeutics. Nature Reviews Drug Discovery. [4] Diebold SS, et al. (2004). Science. [5] Roberts TC, et al. (2020). Nature Reviews Drug Discovery.

Other names

Single-stranded RNAss-RNAMessenger RNAmRNAViral RNA genomeNon-coding RNA

Sequence-specific hybridization leading to RNase H-mediated degradation, RNA interference (RNAi) via the RISC complex, modulation of pre-mRNA splicing patterns, or inhibition of viral RNA-dependent RNA polymerase (RdRp) activity through chain termination.