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Updated: May 23, 2026
09:51
Establishment of Genome-edited Human Pluripotent Stem Cell Lines: From Targeting to Isolation
Published on: February 2, 2016
Genome editing in induced pluripotent stem cells
Li-Tao Cheng1, Liang-Tso Sun, Takashi Tada
1Stem Cell Engineering, Institute for Frontier Medical Sciences, Kyoto University, Kyoto 606-8507, Japan.
Genes to Cells : Devoted to Molecular & Cellular Mechanisms
|April 11, 2012
Summary
Induced pluripotent stem (iPS) cells offer regenerative medicine potential. Genome editing advances iPS cell therapy for genetic disorders by correcting mutations, enabling clinical applications.
Area of Science:
- Stem cell biology
- Genetics
- Regenerative medicine
Background:
- Induced pluripotent stem (iPS) cells hold promise for regenerative medicine by generating patient-specific cells and tissues.
- Therapeutic applications of iPS cells for genetic disorders require efficient and specific genome-editing technologies.
Purpose of the Study:
- To review the application of genome-editing technologies in induced pluripotent stem cells.
- To highlight the potential of genome-edited iPS cells for treating genetic disorders.
Main Methods:
- Review of recent genome-editing strategies, including zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), applied to human and mouse iPS cells.
- Discussion of spontaneous homologous recombination for correcting genetic mutations in iPS cells.
Main Results:
- Genome-editing technologies have been successfully applied to human and mouse iPS cells.
- Spontaneous homologous recombination can correct genetic mutations in iPS cells.
Conclusions:
- Genome editing is crucial for realizing the clinical potential of patient-specific, mutation-free iPS cells in treating genetic disorders.
- Advancements in genome editing pave the way for iPS cell-based therapies for a range of genetic conditions.
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01:06Induced Pluripotent Stem Cells
Stem cells are undifferentiated cells that divide and produce different cell types. Ordinarily, cells that have differentiated into a specific cell type are terminally differentiated; however, scientists have found a way to reprogram these mature cells so that they dedifferentiate and return to an unspecialized, proliferative state. These cells are pluripotent like embryonic stem cells—able to produce all cell types—and are called induced pluripotent stem cells (iPSCs).
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Stem cells are undifferentiated cells that divide and produce different types of cells. Ordinarily, cells that have differentiated into a specific cell type are post-mitotic—that is, they no longer divide. However, scientists have found a way to reprogram these mature cells so that they “de-differentiate” and return to an unspecialized, proliferative state. These cells are also pluripotent like embryonic stem cells—able to produce all cell types—and are therefore called induced pluripotent stem...
01:32Chromatin Modification in iPS Cells
Chromatin modification alters gene expression; therefore, scientists can add histone-modifying enzymes, histone variants, and chromatin remodeling complexes to somatic cells to aid reprogramming into pluripotent stem (iPS) cells.
Compact chromatin makes reprogramming difficult. Enzymes, such as histone demethylases and acetyltransferases, are often added during reprogramming to loosen the chromatin, making the DNA more accessible to transcription factors. Molecules that inhibit histone...
Compact chromatin makes reprogramming difficult. Enzymes, such as histone demethylases and acetyltransferases, are often added during reprogramming to loosen the chromatin, making the DNA more accessible to transcription factors. Molecules that inhibit histone...
01:29Somatic to iPS Cell Reprogramming
Reprogramming alters the gene expression in somatic cells, transforming them into induced pluripotent stem (iPS) cells over several generations. Scientists can reprogram cells by introducing genes for four transcription factors—Oct4, Sox2, Klf4, and c-Myc (OSKM) by viral or non-viral methods. These factors are also known as Yamanaka factors after Shinya Yamanaka, who first generated iPS cells using mouse skin cells. Yamanaka was awarded the Nobel Prize in Physiology or Medicine in 2012 for this...