Nucleic acids (DNA and RNA) (DNA/RNA)

Nucleic acids, comprising deoxyribonucleic acid (DNA) and ribonucleic acid (RNA), are the fundamental molecules responsible for the storage, transmission, and expression of genetic information in all living organisms (National Human Genome Research Institute, 2023). DNA serves as the long-term repository of the genome, while various RNA species, including mRNA, tRNA, and rRNA, facilitate the translation of genetic code into functional proteins and provide regulatory control over cellular processes (Nature Education, 2014). In clinical practice, DNA has historically been a primary target for cytotoxic chemotherapies, such as alkylating agents and platinum-based drugs, which induce structural damage to halt the proliferation of malignant cells (PubMed, PMID: 28331195). More recently, RNA has emerged as a versatile therapeutic target through the development of antisense oligonucleotides and RNA interference (RNAi) technologies, which allow for the highly specific silencing of disease-associated genes or the correction of aberrant splicing (Nature Reviews Drug Discovery, 2020). Targeting nucleic acids is a cornerstone of treatment for oncology, viral infections, and rare genetic disorders, though it necessitates careful management of risks such as genotoxicity and off-target immune stimulation (PubMed, PMID: 31558516).

Other names

Deoxyribonucleic acidRibonucleic acidPolynucleotidesGenetic materialGenome

Therapeutic strategies targeting nucleic acids include DNA alkylation and covalent cross-linking to induce apoptosis (StatPearls, 2023), intercalation to inhibit replication and transcription, and the use of sequence-specific antisense oligonucleotides (ASOs) or small interfering RNAs (siRNAs) to degrade target mRNA or modulate splicing patterns (Nature Reviews Drug Discovery, 2020).