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Updated: Jan 6, 2026
09:04
Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells
Published on: September 25, 2019
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CRISPR-Based Functional Genomics in Pluripotent Stem Cells
1Department of Medicine and Biomedical Sciences, University of Algarve, Faro, Portugal. zahedisetareh@gmail.com.
Stem Cell Reviews and Reports
|November 14, 2025
Summary
CRISPR gene editing combined with pluripotent stem cells (PSCs) revolutionizes gene function studies and disease modeling. This powerful framework accelerates biological discovery and clinical translation for regenerative medicine.
Area of Science:
- Biotechnology
- Genomics
- Stem Cell Biology
Background:
- CRISPR-Cas technology and pluripotent stem cells (PSCs) are key tools in modern biology.
- Their integration offers unprecedented opportunities for gene function analysis and disease modeling.
Purpose of the Study:
- To comprehensively review recent advancements in CRISPR-Cas platforms applied to PSCs.
- To evaluate methodologies for genome-wide screening, lineage tracing, and therapeutic engineering using human PSCs.
Main Methods:
- Review of studies utilizing human embryonic stem cells and induced pluripotent stem cells with CRISPR-Cas systems.
- Analysis of CRISPR-based gene knockouts, base editing, prime editing, and CRISPR activation/interference (CRISPRa/i).
Main Results:
- CRISPR-PSC integration shows progress in editing efficiency, delivery, and safety.
- Limitations include off-target effects, epigenetic variability, and cell-specific responses.
- Successful applications include generating immune-evasive PSCs and developing organoid models.
Conclusions:
- CRISPR-PSC functional genomics is a versatile framework for understanding human biology and disease.
- It accelerates the translation of research findings into clinical applications.
- Further refinement is needed to overcome existing limitations for broader therapeutic use.
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