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Updated: Jun 11, 2026

CRISPR Epigenome Editing in Human Cells using Plasmid DNA Transfection and mRNA Nucleofection Delivery
Published on: May 30, 2025
Next generation technologies for CRISPR-based epigenome and transcriptional modulation
Rithu K Pattali1, Nikita S Divekar1, James K Nuñez1,2
1Department of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA 94720, USA.
Epigenome editing and transcriptional modulation tools, including CRISPR, offer precise gene control without DNA breaks. These advancements are revolutionizing research and hold promise for new therapies.
Area of Science:
- Molecular Biology
- Genetics
- Epigenetics
Background:
- Epigenetic modification and transcription control technologies are crucial for gene perturbation and functional genomics.
- Epigenome editing provides a safe and precise method for altering gene expression by avoiding DNA breaks.
- CRISPR technology has significantly advanced the development of tools for programmable epigenetic and transcriptional modulation.
Purpose of the Study:
- To review recent engineering of novel tools for epigenome editing and transcriptional modulation.
- To discuss the therapeutic potential of epigenome modulators.
- To highlight challenges in improving the safety and efficacy of these technologies.
Main Methods:
- Leveraging rational design, high-throughput screening, and mutational scans to engineer novel tools.
- Utilizing the endogenous reservoir of chromatin and transcriptional effectors.
- Targeted gene repression and activation strategies.
Main Results:
- Significant discoveries in engineering novel epigenome editing and transcriptional modulation tools.
- Demonstration of targeted gene repression and activation through engineered tools.
- Identification of therapeutic potential for epigenome modulators.
Conclusions:
- Advancements in epigenome editing and transcriptional modulation are revolutionizing basic research and functional genomics.
- Epigenome modulators show promise for therapeutic applications, but safety and efficacy challenges remain.
- Further understanding of gene expression mechanisms is key to developing next-generation technologies for research and translation.
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