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Can genome engineering be used to target cancer-associated enhancers?

Matthew R Grimmer1, Peggy J Farnham

  • 1Norris Comprehensive Cancer Center, Keck School of Medicine, University of Southern California, Los Angeles, CA 90089-9601, USA.

Epigenomics
|November 29, 2014
PubMed

Insights

Transcriptional misregulation drives diseases like cancer. Targeting cell-specific enhancers offers a precise therapeutic strategy to restore normal gene regulation with potentially fewer side effects than broad epigenetic drugs.

Area of Science:

  • Genetics
  • Epigenetics
  • Molecular Biology

Background:

  • Transcriptional misregulation is a key factor in disease development, particularly in neoplastic transformation.
  • Cell type-specific enhancers are crucial for controlling gene expression patterns in normal and diseased tissues.
  • Enhancers represent promising targets for novel therapeutic interventions.

Purpose of the Study:

  • To explore the potential of targeting cell type-specific enhancers for therapeutic intervention.
  • To investigate methods for precise genome engineering at enhancer regions.
  • To highlight the need for increased specificity and efficiency in enhancer-targeting technologies.

Main Methods:

  • Utilizing genomic nucleases for targeted modifications.
  • Employing site-specific epigenetic regulators.
  • Leveraging artificial DNA-binding proteins and RNA-DNA interactions for precise targeting.

Main Results:

  • Demonstrated the role of enhancers in cell-specific gene regulation.
  • Showcased potential for precise genome engineering at enhancers.
  • Identified the early stage of development for this therapeutic approach.

Conclusions:

  • Modulating gene regulation via cell type-specific enhancers offers a promising therapeutic avenue.
  • Precise genome engineering at enhancers could restore normal epigenetic patterns with reduced side effects.
  • Further advancements in specificity and efficiency are crucial for clinical application.
Keywords:
CRISPRsDNA methylationTALENsZFNsenhancersepigenetic therapygene expressiongenome engineeringgenomic nucleasehistone modifications

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