iPSC Diseases Model with CRISPR/Cas9


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iPSC Diseases Model with CRISPR/Cas9    

In the past decade, induced pluripotent stem cells (iPS) have revolutionized human disease models. The establishment of iPSCs as a useful method for studying different diseases suggests a clearer reflection of certain aspects of the human genetic defect disease phenotype rather than rodent models. At the same time, iPS-derived and differentiated cells enable researchers to study the effects of different cell types on health and disease, as well as biomarkers and therapeutic drug screening in the human genetic context.

Recently, the combination of induced pluripotent stem cells (iPSCs) and CRISPR technology has made a breakthrough in disease analysis and modeling. The simplicity and versatility of CRISPR are ideal for simulating many different diseases because it allows complex manipulation of genes in impossible ways. The functional knock-out model of PKD shows features such as cyst formation and podocyte tissue loss and cell junction integrity in iPSC-derived organoids. This has also led to an in vitro system that is easy to study nephrotoxicity, which is the most important obstacle in the design and testing of new drugs. In 2016, the CRISPR-Cas9 system was first used to mimic AD mutations, more faithfully mimicking the pathology of AD patients, and enhancing our understanding of the pathogenesis of these important diseases.

Generation of edited iPSCs and clinical applications Figure 1: Generation of edited iPSCs and clinical applications (Eun Ji Ki. 2017)

CRISPR / Cas9 PlatformCB, a global leading genetic editing biotechnology company, is committed to providing the most professional and comprehensive genetic editing technology solutions for our clients working on the editing of CRISPR/Cas9 genes. To support your projects, we offer a comprehensive custom CRISPR/Cas9 gene editing service from strategy design to final stable cell line. We have successfully obtained >500 unique iPSC models with a success rate of > 98%, which have recognized by our customers. In addition, we will provide you with a final report, which details each step, including target vector design, construction, and validation, transfection conditions, genotyping strategies and results. We also provide high-quality products, including CRISPR/Cas9 related kits, Cas9 enzyme. If you have any questions, please feel free to contact us.

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  2. Vo Van Giau. et al. Genome-editing applications of CRISPR–Cas9 to promote in vitro studies of Alzheimer’s disease. Clin Interv Aging. 2018; 13:221–233.
  3. Freiermuth, J. L. et al. Toward a CRISPR Picture: Use of CRISPR/Cas9 to Model Diseases in Human Stem Cells in Vitro. Journal of Cellular Biochemistry. 2017; 119(1): 62–68.
  4. Liquan Cai. et al. CRISPR-mediated genome editing and human diseases. Genes & Disease. 2016; 3(4): 244-251.
  5. Eun Ji Kim. et al. CRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells. Korean J Intern Med. 2017; 32(1):42-61.
For research use only. Not intended for any clinical use.