RNA metabolism enzymes and RNA processing machinery

RNA metabolism enzymes and RNA processing machinery represent a broad class of proteins involved in the synthesis, modification, and degradation of RNA (Nature Reviews Molecular Cell Biology, 2018). This machinery includes RNA polymerases, the spliceosome, RNA helicases, and various exonucleases that ensure the fidelity and regulation of the transcriptome (Cell, 2020). Dysregulation of these processes is a hallmark of several diseases; for instance, mutations in spliceosomal components like SF3B1 are frequent in myeloid leukemias, while defects in RNA-binding proteins are linked to neurodegenerative conditions such as Amyotrophic Lateral Sclerosis (ALS) (Nature Reviews Cancer, 2021; Neuron, 2017). Therapeutic intervention in this space has seen significant success with the development of splicing modifiers like Risdiplam for Spinal Muscular Atrophy and RNA polymerase inhibitors for viral infections (New England Journal of Medicine, 2020). However, because these enzymes are often essential for general cell survival, achieving a therapeutic window without causing global transcriptomic disruption remains a primary challenge in drug development (Trends in Pharmacological Sciences, 2019). The field is currently expanding toward targeting specific RNA-protein interactions and utilizing antisense oligonucleotides to correct processing errors in rare genetic diseases.

Other names

RNA processing factorsRNA metabolic machineryRNA-binding proteins (RBPs)Spliceosome componentsRNA-modifying enzymes

Modulation of pre-mRNA splicing, inhibition of RNA-dependent RNA polymerase (RdRp), inhibition of DNA-directed RNA polymerase, and interference with RNA processing and stability.