Target intelligence / Profile preview

Nucleic acid (DNA and RNA) (DNA/RNA)

Target
DNA/RNA
Molecular classification
Nucleic acid, Biopolymer
01

Overview

Nucleic acids, encompassing deoxyribonucleic acid (DNA) and ribonucleic acid (RNA), are the primary molecules responsible for the storage, transmission, and expression of genetic information in all living organisms. DNA is predominantly located in the nucleus, where it serves as the permanent template for life, while various forms of RNA operate in both the nucleus and cytoplasm to translate genetic codes into functional proteins and regulate cellular processes [1][3]. In pharmacology, these molecules are critical therapeutic targets; for instance, traditional chemotherapies like alkylating agents and intercalators directly damage DNA to induce apoptosis in rapidly dividing cancer cells [4]. More recently, the therapeutic landscape has expanded to include RNA-targeted therapies, such as antisense oligonucleotides and siRNA, which selectively modulate the expression of disease-associated proteins [2]. While highly effective, targeting nucleic acids poses significant challenges, including the risk of non-specific genomic damage and the potential for long-term mutagenic effects [5]. Sources: [1] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3175321/ [2] https://www.nature.com/articles/nrd.2017.101 [3] https://www.uniprot.org/keywords/KW-0540 [4] https://www.cancer.gov/about-cancer/treatment/types/chemotherapy [5] https://pubmed.ncbi.nlm.nih.gov/21531079/

Other names
Deoxyribonucleic acidRibonucleic acidGenetic materialPolynucleotideGenomic DNAMessenger RNANuclear DNACytoplasmic RNA
02

Mechanism of action

Drugs interact with nucleic acids through various mechanisms including covalent cross-linking (alkylation), intercalation between base pairs, inhibition of synthesis via antimetabolites, induction of strand breaks through topoisomerase inhibition, and sequence-specific binding via antisense oligonucleotides or RNA interference (RNAi) to modulate translation or degradation [1][2].

03

Biological functions

Genetic information storageProtein synthesisGene expression regulationSignal transductionCatalysis
04

Disease associations

CancerInfectionGenetic disorderAutoimmune diseaseInflammation
05

Safety considerations

GenotoxicityMutagenicitySecondary malignanciesMyelosuppressionOff-target gene silencingInnate immune activation
06

Interacting drugs

Cisplatin

7 more in the full profile.

07

Biomarkers

Circulating tumor DNA (ctDNA)DNA mutation status (e.g., BRCA1/2)RNA expression profilesMicrosatellite instability (MSI)Tumor mutational burden (TMB)

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