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Updated: Mar 30, 2026

CRISPR Epigenome Editing in Human Cells using Plasmid DNA Transfection and mRNA Nucleofection Delivery
Published on: May 30, 2025
Controlling transcription in human pluripotent stem cells using CRISPR-effectors.
Ryan M Genga1, Nicola A Kearns1, René Maehr1
1Program in Molecular Medicine, Diabetes Center of Excellence, University of Massachusetts Medical School, Worcester, MA 01605, USA.
CRISPR-effector systems enable precise control over gene transcription in human pluripotent stem cells (hPSCs). These tools accelerate the discovery of genes and mechanisms driving cell differentiation and state.
Area of Science:
- Stem cell biology
- Molecular biology
- Gene editing technologies
Background:
- Human pluripotent stem cells (hPSCs) are crucial for studying cellular development and disease.
- Manipulating transcription in hPSCs is key to understanding cell fate and differentiation.
- CRISPR-effector systems offer powerful tools for gene function analysis.
Purpose of the Study:
- To review recent advances in CRISPR-effector technologies for transcriptional control.
- To highlight the application of these systems in human pluripotent stem cells.
- To demonstrate the effectiveness of CRISPR-mediated transcriptional regulation.
Main Methods:
- Review of CRISPR-effector technologies for gene activation, repression, and epigenome engineering.
- Application of CRISPR-effector systems for transcriptional regulation in hPSCs.
- Targeting a synthetic promoter driving a GFP transgene to assess CRISPR function.
Main Results:
- CRISPR-effector systems provide a versatile platform for controlling gene transcription.
- These systems are effective and easy to implement in hPSCs.
- Demonstrated successful transcriptional regulation via a synthetic promoter driving GFP.
Conclusions:
- CRISPR-effector technologies represent a significant advancement for functional genomics in hPSCs.
- These tools facilitate rapid gene function discovery and mechanistic insights.
- CRISPR-mediated transcriptional control offers a promising approach for stem cell research.
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