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Updated: Aug 30, 2026

In Vitro Selection of Engineered Transcriptional Repressors for Targeted Epigenetic Silencing
10:44

In Vitro Selection of Engineered Transcriptional Repressors for Targeted Epigenetic Silencing

Published on: May 5, 2023

Reactivation of silenced genes and transcriptional therapy

P Chiurazzi1, G Neri

  • 1Institute of Medical Genetics, Catholic University, Rome, Italy.

Cytogenetic and Genome Research
|October 4, 2003
PubMed

Insights

Transcriptional therapy offers a promising approach to treat genetic disorders by modulating gene expression. This review explores targeting DNA methylation and histone acetylation for therapeutic gene regulation.

Area of Science:

  • Molecular Biology
  • Epigenetics
  • Genetics

Background:

  • Gene expression is crucial for cellular function and development.
  • Aberrant gene silencing, through DNA methylation or histone modifications, underlies various genetic disorders.
  • Current research explores epigenetic modifications as therapeutic targets.

Purpose of the Study:

  • To review the potential of transcriptional therapy for treating genetic disorders.
  • To categorize target genes based on their epigenetic status and silencing mechanisms.
  • To discuss advances in chromatin regulation relevant to gene therapy.

Main Methods:

  • Categorization of target genes into four groups based on methylation and acetylation status.
  • Review of in vitro and in vivo experimental data using epigenetic drugs.
  • Discussion of chromatin regulation mechanisms, including histone methylation and RNA interference.

Main Results:

  • Transcriptional therapy aims to restore normal gene expression.
  • DNA demethylating and histone hyperacetylating drugs are being investigated.
  • Understanding chromatin regulation is key to developing effective gene therapies.

Conclusions:

  • Transcriptional therapy holds significant potential for treating monogenic and multifactorial disorders.
  • Future advancements in transcription regulation could lead to effective genetic treatments.
  • Epigenetic modifications represent a viable therapeutic strategy for genetic conditions.

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