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Updated: Nov 1, 2025
07:49
CRISPR Epigenome Editing in Human Cells using Plasmid DNA Transfection and mRNA Nucleofection Delivery
Published on: May 30, 2025
1.7K
Controlling CRISPR with small molecule regulation for somatic cell genome editing
Namita Khajanchi1, Krishanu Saha2
1Department of Biomedical Engineering, University of Wisconsin-Madison, Madison, WI 53706, USA.
Summary
Small molecules offer precise control over CRISPR-Cas gene editing systems, enhancing safety and efficiency for in vivo somatic cell therapies by minimizing off-target effects and adverse events.
Area of Science:
- Biotechnology
- Genetics
- Molecular Biology
Background:
- CRISPR-Cas systems enable programmable gene editing but can cause genotoxicity and adverse events due to off-target cleavage.
- Translational challenges for in vivo somatic cell editing include viral vector limitations and host immune responses.
Purpose of the Study:
- To review strategies for controlling CRISPR-Cas systems in vivo.
- To focus on small molecules as regulatory switches for Cas genome editors.
Main Methods:
- Review of current strategies for conditional control of Cas systems.
- Focus on small molecule-based regulatory mechanisms.
Main Results:
- Small molecules, light, magnetism, and temperature are used to control Cas system activity.
- Small molecules can be incorporated as regulatory switches.
Conclusions:
- Small molecule regulation of CRISPR-Cas editors holds significant potential for improving in vivo somatic cell genome editing.
- This approach can increase editing efficiency while reducing adverse effects.
Keywords:
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