Transcriptome

The transcriptome is the complete set of all RNA molecules, including messenger RNA (mRNA), transfer RNA (tRNA), ribosomal RNA (rRNA), and various non-coding RNAs, produced by the genome of a cell or a population of cells (NHGRI, 2024). Unlike the relatively static genome, the transcriptome is highly dynamic and reflects the real-time physiological state, developmental stage, and environmental responses of an organism (Wang et al., 2009, Nature Reviews Genetics). While the transcriptome itself is a collective entity rather than a discrete therapeutic target, its analysis is foundational for identifying specific RNA-based targets and understanding the broader molecular mechanisms of drug action (Casamassimi et al., 2017, International Journal of Molecular Sciences). In clinical research, transcriptomic profiling is widely used to identify biomarkers for disease diagnosis, prognosis, and the assessment of therapeutic efficacy or toxicity (Lowe et al., 2017, Nature Reviews Genetics). By analyzing changes in the transcriptome, researchers can gain insights into the aberrant pathways driving complex diseases such as cancer and autoimmune disorders, facilitating the development of precision medicine (Ozsolak & Milos, 2011, Nature Reviews Genetics). Consequently, the transcriptome serves as an indispensable resource for drug discovery and patient stratification, even though it is not a target in the classical pharmacological sense.

Other names

RNA profileGene expression profileTotal RNA transcriptsExome (RNA)

Not applicable; the transcriptome is a collective set of RNA molecules rather than a specific molecular target for drug binding.