A precise therapeutic strategy involving the use of specific compounds or environmental factors to modulate the expression level of particular genes within target cells or tissues. This regulation can involve upregulating beneficial genes, such as those involved in DNA repair or antioxidant defense, or downregulating genes associated with disease pathways, like inflammation or fibrosis. It represents a molecular-level approach to influencing physiological outcomes.
Origin
This term is rooted in the field of molecular genetics and pharmacology, leveraging the understanding of transcription factors and epigenetic mechanisms. Targeted emphasizes the specificity of the intervention to a select gene or gene family, moving beyond broad-spectrum effects. The goal is to influence the proteome and therefore the function of the cell.
Mechanism
Regulation can be achieved through small molecules that bind to nuclear hormone receptors, altering their ability to interact with DNA response elements, or through nutritional compounds that act as epigenetic modulators, influencing DNA methylation or histone acetylation. For instance, specific compounds can enhance the transcription of genes that increase estrogen receptor beta expression. This precise control over gene expression allows for highly individualized therapeutic effects.
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