Ribonucleic acid transcript (RNA)

Ribonucleic acid (RNA) transcripts are the primary products of gene transcription, serving as the essential link between genomic DNA and the proteome. This broad class includes messenger RNA (mRNA), which encodes proteins, and various non-coding RNAs (ncRNAs) that regulate cellular processes (National Center for Biotechnology Information [NCBI], 2024). In modern drug discovery, RNA transcripts are targeted to address proteins that were previously considered undruggable by intervening at the pre-translational level (Nature Reviews Drug Discovery, 2017). Therapeutic strategies such as antisense oligonucleotides (ASOs) and RNA interference (RNAi) enable the sequence-specific degradation of toxic transcripts or the correction of aberrant splicing patterns (PubMed, 2019). Additionally, the development of small molecules targeting RNA secondary structures has expanded the scope of RNA-targeted therapies (Science, 2020). These approaches are currently utilized to treat a variety of conditions, including genetic disorders like spinal muscular atrophy, hereditary amyloidosis, and certain types of muscular dystrophy (FDA, 2023).

Other names

Ribonucleic acidRNAmRNAMessenger RNApre-mRNANon-coding RNATranscriptome

Therapeutic targeting of RNA transcripts involves several distinct modalities: antisense oligonucleotides (ASOs) that induce RNase H-mediated degradation or modulate splicing (e.g., Nusinersen) (Biogen, 2016); small interfering RNAs (siRNAs) that engage the RNA-induced silencing complex (RISC) to degrade target mRNA (e.g., Patisiran) (Alnylam Pharmaceuticals, 2018); and small molecules that bind to RNA structural motifs to interfere with translation or splicing (e.g., Risdiplam) (Roche, 2020). These mechanisms allow for the precise down-regulation of disease-causing proteins or the restoration of functional protein production (Nature Reviews Drug Discovery, 2017).