Epigenome editing technologies for discovery and medicine

Sean R McCutcheon1,2, Dahlia Rohm1,2, Nahid Iglesias1,2

  • 1Department of Biomedical Engineering, Duke University, Durham, NC, USA.

Nature Biotechnology
|July 29, 2024
PubMed
Summary

Epigenome editing uses CRISPR technology to precisely alter gene expression without DNA breaks. This powerful approach holds significant potential for understanding gene regulation and treating complex diseases.

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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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Epigenetic changes alter the physical structure of the DNA without changing the genetic sequence and often regulate whether genes are turned on or off. This regulation ensures that each cell produces only proteins necessary for its function. For example, proteins that promote bone growth are not produced in muscle cells. Epigenetic mechanisms play an essential role in healthy development. Conversely, precisely regulated epigenetic mechanisms are disrupted in diseases like cancer.
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