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Endogenous Protein Tagging in Human Induced Pluripotent Stem Cells Using CRISPR/Cas9
Published on: August 25, 2018
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Genome Editing in Induced Pluripotent Stem Cells using CRISPR/Cas9.
Ronen Ben Jehuda1,2,3,4, Yuval Shemer1,2,3, Ofer Binah5,6,7
1Department of Physiology, Biophysics and Systems Biology, Rappaport Faculty of Medicine, Technion, 1 Efron Street, POB 9649, 31096, Haifa, Israel.
Stem Cell Reviews and Reports
|April 7, 2018
Summary
Induced Pluripotent Stem Cells (iPSC) and CRISPR/Cas9 gene editing are revolutionizing inherited disease research. This review explores how combining these technologies aids in understanding disease mechanisms by creating precise cellular models.
Area of Science:
- Stem Cell Biology and Regenerative Medicine
- Genetics and Genomics
- Disease Modeling
Background:
- Induced Pluripotent Stem Cells (iPSC) technology allows somatic cell reprogramming for disease modeling.
- iPSC-derived cells are crucial for investigating inherited disease mechanisms.
- A key challenge is distinguishing mutation effects from genetic background variations.
Purpose of the Study:
- To review recent advancements in combining iPSC and CRISPR/Cas9 technologies.
- To highlight their application in studying molecular and cellular mechanisms of inherited diseases.
- To emphasize the generation of isogenic controls for accurate disease pathology investigation.
Main Methods:
- Utilizing CRISPR/Cas9 genome editing for precise gene modification in iPSCs.
- Generating isogenic cell lines (mutated vs. control) from iPSCs.
- Differentiating iPSCs into various cell types for disease-specific studies.
Main Results:
- CRISPR/Cas9 enables high-efficiency, accurate gene editing in iPSCs.
- This facilitates the creation of isogenic cell lines, isolating mutation effects.
- Combined technologies are applied across diverse inherited diseases including immunological, metabolic, hematological, neurodegenerative, and cardiac conditions.
Conclusions:
- The synergy of iPSC and CRISPR/Cas9 technologies provides powerful tools for inherited disease research.
- This approach overcomes previous limitations in disease modeling by controlling genetic background.
- Future research will benefit from these precise cellular models to elucidate complex disease pathologies.
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