is this related with cripir improving single gene disorder

Question

he 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

Yes, this is related to using CRISPR technology to improve single gene disorders. The article discusses how CRISPR/Cas9 technology and induced pluripotent stem cells are being used to study human genetics and diseases, and how ASC is using CRISPR to engineer iPSCs to generate isogenic control-disease cell line models for gene therapy in regenerative medicine. This technology allows for precise manipulation of genes related to single gene disorders, providing a new approach for potential treatments.