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Precise Epigenome Editing on the Stage: A Novel Approach to Modulate Gene Expression
1Medical Center - University of Freiburg, Institute for Transfusion Medicine and Gene Therapy, Freiburg, Germany.
Epigenetics Insights
|December 22, 2018
Summary
Designer epigenome modifiers (DEM) offer precise epigenetic editing in human cells without DNA double-strand breaks. This novel platform shows high efficiency and a favorable safety profile, positioning it for clinical translation in treating genetic disorders.
Area of Science:
- Molecular Biology
- Genetics
- Epigenetics
Background:
- Genetic and epigenetic factors significantly contribute to human diseases.
- Precise genomic targeting offers potential cures for genetic disorders.
- Current genome editing technologies face safety concerns, particularly off-target effects.
Purpose of the Study:
- To introduce and evaluate a novel epigenome editing platform, the designer epigenome modifier (DEM).
- To assess the efficiency and safety of DEM for therapeutic applications.
- To explore epigenome editing as a safer alternative to genome editing.
Main Methods:
- Development of a novel platform (DEM) for precise epigenome editing.
- Tethering effectors to DNA-binding moieties for targeted genomic, transcriptomic, or epigenomic alterations.
- Testing DEM in clinically relevant primary human cells.
Main Results:
- DEM enables precise epigenome editing.
- The platform demonstrates high efficiency in primary human cells.
- DEM exhibits a remarkable safety profile, avoiding DNA double-strand breaks.
Conclusions:
- Designer epigenome modifier (DEM) represents a promising technology for epigenome editing.
- DEM's efficiency and safety profile make it a strong candidate for clinical translation.
- Epigenome editing offers a potentially safer therapeutic strategy compared to traditional genome editing.
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