Question

from this figure 17 idnetify the following crispr and single gene disordr how how it is getting impoved

The arrival of CRISPR/Cas9 technology and induced pluripotent stem cells (iPSCs) in recent years has expanded the horizons for gene manipulation in human cells. These two technologies have provided a new arena to study human biology and diseases and are paving a sure path toward gene therapy in regenerative medicine. They provide an unlimited resource of in vitro models of human genetics and diseases.

Applied StemCell () is a premier licensee of both CRISPR/Cas9 (The Broad Institute, MIT) and the Yamanaka-iPSC reprogramming (from iPS Academia, Japan) technologies. It uses CRISPR to engineer iPSCs derived from various clinical sources, to generate isogenic control-disease cell line models that provide reliable comparative results without genetic background variability.

ASC’s CRISPR protocol is well-optimised to: 1) culture and maintain iPSCs without differentiation in a feeder-free culture system; 2) balance transfection of CRISPR elements while maintaining cell viability; 3) uses validated gRNAs to increase Cas9 cutting efficiency and limit off-target modifications; 4) includes a transient expression selection marker to increase the targeting efficiency of CRISPR in iPSCs (Figure 17).

Answer 1

[AI-generated answer — Bot GPT 3.5]

In Figure 17, the CRISPR technology is being used to engineer iPSCs derived from various clinical sources in order to generate isogenic control-disease cell line models. This technology is being applied to study human genetics and diseases, with the goal of paving the way for gene therapy in regenerative medicine.

One possible single gene disorder that could be studied and improved through this technology is cystic fibrosis, a genetic disorder that affects the lungs and digestive system. By using CRISPR to edit the gene responsible for cystic fibrosis in iPSCs, researchers can create a diseased cell model to study the disease mechanisms and test potential therapies.

Overall, the use of CRISPR technology in combination with iPSCs allows for precise gene manipulation and the creation of disease models for research purposes. This approach has the potential to lead to the development of targeted gene therapies for a wide range of genetic disorders.