Target intelligence / Profile preview

Genes

Molecular classification
Nucleic acid
01

Overview

Genes are the fundamental physical and functional units of heredity, consisting of specific sequences of DNA that provide the instructions for synthesizing functional molecules, such as proteins or various types of RNA (National Human Genome Research Institute, 2024). In the context of pharmacology and drug discovery, the term 'Genes' is considered too broad and non-specific to be classified as a singular therapeutic target. While medical interventions like gene therapy or CRISPR-based editing target individual, specific genes to correct mutations, the collective term encompasses the entire genome and does not describe a distinct druggable entity like a receptor or enzyme (National Institutes of Health, 2023). Most traditional drugs interact with the protein products of genes rather than the DNA sequences themselves. Because this entry refers to a general biological category rather than a specific, identifiable molecular target, it is flagged as an incorrect or overly general term for drug-target mapping purposes (UniProt, 2024).

Other names
Hereditary unitsGenetic materialCistronsGenomic DNA
02

Mechanism of action

Not applicable as 'Genes' is a broad biological category rather than a specific molecular target for drug binding.

03

Biological functions

Genetic inheritanceProtein synthesis encodingRNA synthesis templateRegulation of cellular phenotype
04

Disease associations

Genetic disordersCancer (via mutation or dysregulation)Hereditary diseasesCongenital malformations
05

Safety considerations

Ethical concerns regarding germline modificationOff-target effects in gene-editing therapiesGenotoxicityUnintended insertional mutagenesis
06

Biomarkers

Single nucleotide polymorphisms (SNPs)Gene expression signaturesCopy number variations (CNVs)

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