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Updated: May 18, 2026
09:51
Establishment of Genome-edited Human Pluripotent Stem Cell Lines: From Targeting to Isolation
Published on: February 2, 2016
Development of nuclease-mediated site-specific genome modification
Stacey E Wirt1, Matthew H Porteus
1Department of Pediatrics, Stanford University School of Medicine, Stanford, CA 94305, USA.
Current Opinion in Immunology
|September 18, 2012
Summary
Genome engineering uses engineered nucleases to fix gene mutations for treating monogenic diseases. Zinc finger nucleases show promise for correcting genetic disorders like primary immunodeficiencies (PIDs).
Area of Science:
- Genetics and Molecular Biology
- Gene Therapy
- Disease Modeling
Background:
- Monogenic diseases result from specific gene mutations.
- Genome engineering offers a potential therapeutic strategy.
- Engineered nucleases can precisely target and correct DNA sequences.
Purpose of the Study:
- To explore the application of engineered nucleases in treating monogenic diseases.
- To highlight the potential of zinc finger nucleases for gene correction.
- To discuss the use of nucleases in creating disease models.
Main Methods:
- Utilizing engineered nucleases, such as zinc finger nucleases.
- Employing homologous recombination for gene targeting.
- Applying these techniques in human cells and animal models.
Main Results:
- Demonstrated correction of disease-causing mutations in human cells.
- Successful creation of animal models for human genetic diseases.
- Validated the potential of engineered nucleases for therapeutic applications.
Conclusions:
- Engineered nucleases are a promising therapeutic tool for monogenic diseases.
- Zinc finger nucleases can be designed for targeted gene correction.
- Further clinical development is needed, but potential for primary immunodeficiencies (PIDs) is significant.
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