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Updated: Dec 6, 2025

High-throughput Screening for Chemical Modulators of Post-transcriptionally Regulated Genes
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Modulating gene regulation to treat genetic disorders

Navneet Matharu1,2,3, Nadav Ahituv4,5

  • 1Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA. Navneet.Matharu@ucsf.edu.

Nature Reviews. Drug Discovery
|October 6, 2020
PubMed
Summary

Cis-regulation therapy (CRT) uses gene-editing tools to control gene expression, offering a new way to treat diseases caused by abnormal gene dosage. This review explores CRT technologies and their clinical potential.

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