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CRISPR01:59

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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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RNA Editing02:23

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RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
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Related Experiment Video

Updated: Jan 10, 2026

CRISPR Epigenome Editing in Human Cells using Plasmid DNA Transfection and mRNA Nucleofection Delivery
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CRISPR Epigenome Editing in Human Cells using Plasmid DNA Transfection and mRNA Nucleofection Delivery

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Epigenetic editing: from concept to clinic.

Elizabeth A Heller1, Lacramioara Bintu2, Marianne G Rots3

  • 1Department of Systems Pharmacology and Translational Therapeutics, University of Pennsylvania, Philadelphia, PA, USA.

Nature Reviews. Drug Discovery
|November 25, 2025
PubMed
Summary
This summary is machine-generated.

Epigenetic editing offers a novel therapeutic approach by reprogramming gene expression without altering the genome. Despite early challenges, ongoing research is enhancing its efficacy and specificity for treating diseases.

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Area of Science:

  • Biochemistry
  • Genetics
  • Molecular Biology

Background:

  • Dysregulated gene expression underlies most human diseases.
  • Epigenetic modifications are key mediators of gene expression.
  • Epigenetic editing offers a way to modulate gene expression without genomic alteration.

Purpose of the Study:

  • To review the progress and challenges in epigenetic editing.
  • To highlight the therapeutic potential of epigenetic editing.
  • To discuss future directions for advancing epigenetic editing technologies.

Main Methods:

  • Review of existing literature on epigenetic editing.
  • Analysis of successes in animal models and early clinical trials.
  • Discussion of mechanistic understanding and technological advancements.

Main Results:

  • Epigenetic editing has shown promise in animal models for various diseases.
  • The first clinical trials for epigenetic editing have commenced.
  • Increased understanding of epigenetic reprogramming is addressing initial efficacy and specificity concerns.

Conclusions:

  • Epigenetic editing is evolving into a potent therapeutic strategy.
  • Overcoming challenges in specificity, maintenance, and delivery is crucial for widespread application.
  • Future advancements will solidify epigenetic editing as a powerful therapeutic approach.