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Related Concept Videos

Regulation of Expression at Multiple Steps01:23

Regulation of Expression at Multiple Steps

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The gene expression in cells is regulated at different stages: (i) transcription, (ii) RNA processing, (iii) RNA localization, and (iv) translation. Transcriptional regulation is mediated by regulatory proteins such as transcription factors, activators, or repressors—these control gene expression by initiating or inhibiting the transcription of genes. Once a precursor or pre-mRNA is produced, it undergoes post-transcriptional modification, including 5' capping, splicing, and the...
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Regulation of Expression Occurs at Multiple Steps02:24

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Gene expression can be regulated at almost every step from gene to protein. Transcription is the step that is most commonly regulated. This involves the binding of proteins to short regulatory sequences on the DNA. This association can either promote or inhibit the transcription of a gene associated with the respective sequence.
Transcription results in the generation of precursor (pre-mRNA) that consists of both exons and introns, which needs further processing before being translated to a...
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Regulation of Expression Occurs at Multiple Steps02:24

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Eukaryotic Transcription Inhibitors01:52

Eukaryotic Transcription Inhibitors

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Certain biochemical processes, such as embryonic development and cell growth regulation, depend on the repression of specific genes. DNA binding proteins known as eukaryotic transcription inhibitors regulate the repression of gene expression in eukaryotes. The presence of these inhibitors at the required location and time in the cell is triggered by the presence of hormones and additional signals from other cells.
Eukaryotic transcription inhibitors usually contain two distinct domains, a...
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Co-activators and Co-repressors02:04

Co-activators and Co-repressors

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Gene transcription is regulated by the synergistic action of several proteins that form a complex at a gene regulatory site. This is observed in eukaryotes, where the regulation of gene expression is a complex process. Regulatory proteins in eukaryotes can broadly be classified into two types – regulators that bind directly to specific DNA sequences and co-regulators that associate with regulatory proteins but cannot directly bind to the DNA. These co-regulators are further divided into...
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Updated: Apr 3, 2026

High-throughput Screening for Chemical Modulators of Post-transcriptionally Regulated Genes
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Transcriptional Regulatory Modulation as a Potential Therapeutic Modality.

Navneet Matharu1, Nadav Ahituv1

  • 1Department of Bioengineering and Therapeutic Sciences and Institute for Human Genetics, University of California, San Francisco, California, USA; email: nkmatharu@gmail.com, nadav.ahituv@ucsf.edu.

Annual Review of Genomics and Human Genetics
|April 1, 2026
PubMed
Summary
This summary is machine-generated.

Gene expression modification systems offer therapeutic potential for diseases caused by altered gene levels. These programmable technologies are advancing toward clinical application, addressing delivery and other challenges.

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Last Updated: Apr 3, 2026

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Area of Science:

  • Molecular biology
  • Gene therapy
  • Biotechnology

Background:

  • Human diseases are frequently linked to abnormal gene expression levels.
  • Modulating specific gene expression holds significant therapeutic promise for various conditions.

Purpose of the Study:

  • To review emerging gene expression modification technologies.
  • To assess their therapeutic potential, clinical delivery methods, and associated challenges.

Main Methods:

  • Review of systems utilizing nuclease-deficient gene-editing proteins fused to transcriptional modulators.
  • Targeting of gene regulatory elements to control gene expression.

Main Results:

  • These systems are powerful, programmable, and customizable tools for gene expression modulation.
  • Several such systems are already in clinical use, with many more in development.

Conclusions:

  • Emerging gene editing technologies show considerable therapeutic potential.
  • Further development is needed to overcome clinical delivery and implementation challenges.