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Updated: Feb 13, 2026
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Introducing Point Mutations into Human Pluripotent Stem Cells Using Seamless Genome Editing
Published on: May 10, 2020
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Genome Editing in Stem Cells for Disease Therapeutics
Minjung Song1, Suresh Ramakrishna2,3
1Department of Food Biotechnology, College of Medical and Life Science, Silla University, Busan, South Korea. songmj@silla.ac.kr.
Molecular Biotechnology
|March 9, 2018
Summary
Programmable nucleases like CRISPR enable precise gene editing in stem cells for advanced biological research and therapeutic development. These genome editing tools offer efficient methods for creating disease models and novel treatments.
Area of Science:
- Molecular Biology
- Genetics
- Biotechnology
Background:
- Programmable nucleases are powerful genome editing tools.
- Zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and CRISPR/CRISPR-associated protein (Cas) systems are key examples.
- These nucleases allow precise gene modification.
Purpose of the Study:
- To review the characteristics and mechanisms of major programmable nucleases.
- To explore the applications of these nucleases in stem cell research for disease therapies.
- To summarize key studies in the field of genome editing for therapeutics.
Main Methods:
- Review of scientific literature on programmable nucleases.
- Analysis of mechanisms of action for ZFNs, TALENs, and CRISPR/Cas systems.
- Compilation of studies on genome editing in pluripotent and hematopoietic stem cells.
Main Results:
- Detailed overview of programmable nuclease technologies.
- Demonstration of genome editing applications in stem cells for disease modeling and therapy.
- Highlighting the efficiency and speed of developing novel therapeutics.
Conclusions:
- Programmable nucleases represent a significant advancement in genome editing.
- Applications in stem cells hold immense potential for life sciences and medicine.
- Further research promises efficient disease models and effective therapeutic strategies.
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