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Abstract
CRISPR-based functional genomics combined with pluripotent stem cell technologies is recognized as a transformative approach for investigating gene function and modeling human disease.
- Recent developments in CRISPR-Cas platforms include gene knockouts, base and prime editing, and CRISPR activation or interference.
- Human pluripotent stem cells, such as embryonic stem cells and induced pluripotent stem cells, have been utilized for genome-wide screening and therapeutic engineering.
- Advances have been made in editing efficiency, delivery strategies, and genomic safety, though issues like off-target effects and cell-type-specific responses remain.
- Applications of this technology include creating immune-evasive pluripotent stem cell lines and developing organoid models for studying physiological and pathological conditions.
- These advancements provide a framework for understanding human biology and could facilitate the translation of experimental research into clinical applications.
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