Target intelligence / Profile preview

DNA, RNA, and nucleoproteins

Molecular classification
Nucleic acid, Nucleoprotein, Ribonucleoprotein, Other
01

Overview

DNA, RNA, and nucleoproteins constitute the fundamental molecular components responsible for the storage, transmission, and expression of genetic information in all living organisms [1]. DNA serves as the long-term storage of genetic blueprints, while RNA molecules, including mRNA and rRNA, facilitate the translation of these blueprints into functional proteins [1]. Nucleoproteins are complex assemblies, such as ribosomes and histones, that provide structural support and catalyze essential biological processes like protein synthesis and chromatin packaging [2]. In pharmacology, these molecules are major therapeutic targets; for example, alkylating agents and intercalators target DNA to treat various cancers by inducing apoptosis, while many antibiotics target bacterial ribosomes (nucleoproteins) to inhibit protein synthesis [3, 4]. Recent advancements have introduced RNA-targeted therapies, such as antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs), which allow for the precise modulation of gene expression [5]. However, because these targets are ubiquitous and essential for normal cell function, drugs that interact with them often present significant safety concerns, including genotoxicity, myelosuppression, and the potential for secondary malignancies [6]. Sources: [1] Alberts B, et al. Molecular Biology of the Cell. [2] NIH-NHGRI. [3] PubChem-Cisplatin. [4] StatPearls-Ribosome-targeting antibiotics. [5] Nature Reviews Drug Discovery (2022). [6] FDA Guidance on Genotoxicity.

Other names
Nucleic acidsGenetic materialRibonucleoproteinsDeoxyribonucleoproteinsChromatinRibosome
02

Mechanism of action

Drugs targeting these molecules act through various mechanisms including DNA alkylation, intercalation, inhibition of topoisomerases, ribosomal protein synthesis inhibition, and RNA interference or antisense modulation [3, 4, 5].

03

Biological functions

Genetic information storageTranscriptionTranslationDNA replicationGene regulationOther
04

Disease associations

CancerInfectionGenetic disorderAutoimmune diseaseOther
05

Safety considerations

GenotoxicityMyelosuppressionSecondary malignanciesTeratogenicityOff-target gene silencingInfusion-related reactions
06

Interacting drugs

Cisplatin

8 more in the full profile.

07

Biomarkers

Tumor mutational burden (TMB)Microsatellite instability (MSI)Viral load (e.g., HIV-1 RNA)Anti-double-stranded DNA (anti-dsDNA) antibodiesCirculating tumor DNA (ctDNA)

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