Target intelligence / Profile preview

Deoxyribonucleic acid and Ribonucleic acid (DNA and RNA)

Target
DNA and RNA
Molecular classification
Other (Nucleic acid)
01

Overview

Deoxyribonucleic acid (**DNA**) and ribonucleic acid (**RNA**) are the two primary types of nucleic acids found in all living organisms. **DNA** serves as the long-term storage medium for genetic information, encoding instructions necessary for development, functioning, growth, and reproduction. It is typically double-stranded with a deoxyribose sugar backbone and four bases—adenine, guanine, cytosine, and thymine[7][10]. **RNA** is usually single-stranded with a ribose sugar backbone; it contains uracil instead of thymine[7][10]. RNA’s main roles include carrying instructions from DNA to make proteins (*messenger RNA*), forming part of the protein-synthesizing machinery (*ribosomal RNA*), transferring amino acids during protein synthesis (*transfer RNA*), and regulating gene expression (*microRNAs*, *siRNAs*)[2][5].\n\nBoth molecules are essential for life but are not considered therapeutic targets themselves like receptors or enzymes. Instead, they serve as substrates or templates that can be targeted by drugs designed to modulate gene expression or disrupt pathogenic processes at the nucleic acid level. Drugs such as antisense oligonucleotides bind specific sequences on mRNA to inhibit translation; chemotherapeutics may intercalate into DNA strands to prevent cell division.\n\nBecause \"DNA and RNA\" refers collectively to two distinct classes of molecules rather than a single targetable entity—and because neither is a receptor nor an enzyme—the entry is considered incorrect as a therapeutic target under standard drug discovery conventions. Each should be described separately if detailed pharmacological targeting information is required.\n\n> \"The central dogma of molecular biology is 'DNA makes RNA makes protein.' This general rule emphasizes the order of events from transcription through translation...\"[1]\n\n> \"Ribonucleic acid...performs many roles in the cell...carrying biological information...regulating functions...\"[2]

Other names
Nucleic acidsGenetic materialGenome (for DNA)Transcriptome (for RNA)
02

Mechanism of action

Inhibition of gene expression by binding to mRNA or pre-mRNA\n- Intercalation into DNA to disrupt replication/transcription

03

Biological functions

Storage of genetic informationTransmission of genetic informationProtein synthesisGene expression regulation
04

Disease associations

CancerInherited genetic disordersInfectious diseases (as viral genomes)
05

Safety considerations

Off-target effects with nucleic acid-based drugs
06

Interacting drugs

Antisense oligonucleotides (e.g., nusinersen)

2 more in the full profile.

07

Biomarkers

Mutations in specific genes detected in DNA for cancer diagnosis/prognosis

Beyond the preview

Go deeper on Deoxyribonucleic acid and Ribonucleic acid (DNA and RNA).

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Drug pipeline

Full profile access

Explore the programs pursuing this target and their development progress.

  • Drug candidates
  • Developers
  • Development stage

Clinical trials

Full profile access

Follow the clinical studies evaluating therapies directed at this target.

  • Trial design
  • Status
  • Readouts

Competitive landscape

Full profile access

Compare approaches across drug candidates, modalities, and indications.

  • Programs
  • Modalities
  • Indications

Literature & evidence

Full profile access

Investigate the research and source evidence behind target biology and development.

  • Publications
  • Sources
  • Analysis

Patents

Full profile access

Explore patent activity around therapies and technologies addressing this target.

  • Patents
  • Assignees
  • Technologies

Research & analysis

Full profile access

Connect target biology, drug development, and emerging evidence in your research.

  • Biology
  • Development news
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on Deoxyribonucleic acid and Ribonucleic acid (DNA and RNA).

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call