One example of a single gene disorder that could potentially be involved in Figure 17 is cystic fibrosis, which is caused by mutations in the CFTR gene. CRISPR technology has the potential to correct these mutations in patient-derived iPSCs, providing a possible avenue for gene therapy in treating cystic fibrosis.
So, while the specific single gene disorder involved in Figure 17 is not mentioned in the provided information, it is possible that CRISPR technology could be used to improve single gene disorders such as cystic fibrosis.
what is a single gene diorder inolvoled in figure 17
is this related with cripir improving single gene disorder
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).
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