Related Experiment Video
Updated: Apr 20, 2026
09:51
Establishment of Genome-edited Human Pluripotent Stem Cell Lines: From Targeting to Isolation
Published on: February 2, 2016
14.3K
Genome editing in human pluripotent stem cells using site-specific nucleases.
Kunitoshi Chiba1, Dirk Hockemeyer
1Department of Molecular and Cell Biology, University of California, Berkeley, 400B Li Ka Shing Center, Berkeley, CA, 94720-3370, USA.
Methods in Molecular Biology (Clifton, N.J.)
|November 20, 2014
Summary
Human pluripotent stem cells (hPSCs) can now be genetically engineered for targeted gene knockouts and mutations. This advancement significantly aids in studying human diseases within a defined genetic context.
Area of Science:
- Stem cell biology
- Genetic engineering
- Human disease modeling
Background:
- Human embryonic stem cells (hESCs) and induced pluripotent stem cells (iPSCs), collectively human pluripotent stem cells (hPSCs), are vital for disease modeling.
- Genetic engineering in hPSCs was previously limited, unlike in model organisms.
Purpose of the Study:
- To outline protocols for efficient gene targeting in hPSCs.
- To enable precise genetic modifications in pluripotent stem cells for disease research.
Main Methods:
- Utilizing site-specific nucleases for genetic engineering in hPSCs.
- Implementing protocols for targeted gene knockouts, reporter generation, gene overexpression, and mutation introduction/repair.
Main Results:
- Established genetic engineering capabilities in hPSCs, mirroring model systems.
- Enabled targeted gene knockouts, creation of tissue-specific reporters, gene overexpression, and precise mutation management.
- Facilitated the study of human diseases in a controlled genetic environment.
Conclusions:
- Genetic engineering of pluripotent stem cells significantly advances human disease research.
- Protocols for efficient gene targeting in hPSCs are now available.
- Precise genetic control in hPSCs offers new avenues for understanding human pathologies.
