TARGATT™ And CRISPR/Cas9 Modified Induced Pluripotent Stem Cells (iPSCs) For In Vitro Genetic Disease Modeling

Applied StemCell (ASC) showcases its latest innovations in the genetic modification of induced pluripotent stem cells (iPSCs) using CRISPR/Cas9 and its proprietary TARGATT™ technology to advance in vitro modeling of human genetic diseases. The presentation highlights the growing importance of genetically engineered iPSCs as more physiologically relevant systems for understanding disease mechanisms and developing targeted therapies.

ASC provides insights into current trends in gene-modified iPSC research, demonstrating how its complementary platforms — CRISPR for precise gene knockouts and small insertions, and TARGATT™ for efficient integration of large DNA fragments — work together to improve model generation efficiency, precision, and reproducibility.

Key discussion points include strategies for overcoming common challenges in iPSC editing, such as optimizing transfection efficiency, increasing targeting accuracy, and implementing rigorous quality control standards. Real-world case studies of gene knockouts and mutation corrections in both patient- and healthy donor-derived iPSCs further illustrate ASC’s expertise in producing high-quality, disease-relevant cell models.

Together, these technologies provide a robust foundation for accelerating drug discovery, functional genomics, and disease modeling, enabling researchers to generate lineage-specific, genetically defined cellular systems that more accurately represent human biology and therapeutic response.