Target intelligence / Profile preview

Cellular nucleic acids

Molecular classification
Nucleic acid, Other
01

Overview

Cellular nucleic acids, comprising deoxyribonucleic acid (DNA) and various forms of ribonucleic acid (RNA), are the fundamental molecules responsible for the storage, transmission, and expression of genetic information in all living organisms (Alberts et al., Molecular Biology of the Cell, 2014). DNA serves as the stable repository of the genome, while RNA species including mRNA, tRNA, and rRNA facilitate the translation of genetic codes into functional proteins (Lodish et al., Molecular Cell Biology, 2016). These molecules are essential for cellular life, governing processes from replication and transcription to the regulation of complex metabolic pathways. In the context of pharmacology, cellular nucleic acids are critical therapeutic targets for a wide array of diseases, most notably in oncology and infectious diseases. Traditional chemotherapeutic agents, such as cisplatin and doxorubicin, act by directly damaging DNA or inhibiting its replication to halt the proliferation of malignant cells (Pommier et al., Nature Reviews Cancer, 2016). Modern therapeutic strategies have expanded to include sequence-specific RNA-targeted therapies, such as antisense oligonucleotides and small interfering RNAs, which modulate gene expression at the post-transcriptional level to treat genetic disorders (Crooke et al., Nature Reviews Drug Discovery, 2018). However, targeting these ubiquitous molecules presents significant challenges, including the risk of genotoxicity, secondary malignancies, and off-target effects (Goodman & Gilman's, 2018).

Other names
DNARNADeoxyribonucleic acidRibonucleic acidGenetic materialPolynucleotides
02

Mechanism of action

Drugs targeting cellular nucleic acids function through diverse mechanisms including covalent modification (alkylation), non-covalent intercalation, inhibition of metabolic synthesis (antimetabolites), or sequence-specific hybridization (antisense and RNA interference) to disrupt cellular replication and protein production (Goodman & Gilman's, 2018; Crooke et al., 2018).

03

Biological functions

Genetic information storageGene expressionProtein synthesisDNA replicationTranscriptionTranslationGene regulation
04

Disease associations

CancerViral infectionBacterial infectionGenetic disorderOther
05

Safety considerations

GenotoxicityMutagenicitySecondary malignanciesMyelosuppressionOff-target hybridization effectsTeratogenicity
06

Interacting drugs

Cisplatin

7 more in the full profile.

07

Biomarkers

DNA mutation statusRNA expression levelsMicrosatellite instability (MSI)Tumor mutational burden (TMB)

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