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Updated: Apr 3, 2026

High-throughput Screening for Chemical Modulators of Post-transcriptionally Regulated Genes
Published on: March 3, 2015
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.
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.
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.
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