Question

is there any single gnee disordre mentioned? 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]

From the information provided, a single gene disorder that could potentially be mentioned in this context is sickle cell disease. Sickle cell disease is a genetic disorder caused by a mutation in the HBB gene, which codes for the beta globin subunit of hemoglobin.

Using CRISPR/Cas9 technology and induced pluripotent stem cells (iPSCs), researchers can potentially correct the mutation in the HBB gene in patient-derived iPSCs, creating isogenic control-disease cell line models. This approach allows for the study of the disease at a cellular level and the development of potential gene therapies for sickle cell disease.

Improvements in gene therapy for single gene disorders like sickle cell disease can be achieved through advancements in CRISPR/Cas9 technology, such as the optimization of CRISPR protocols to increase efficiency and accuracy of gene editing in iPSCs. This can lead to the development of more effective and targeted gene therapies for single gene disorders.