is there any single gene disorder mentioned in here

Question

Horizon Discovery () is using CRISPR technology to provide solutions at multiple stages of the drug discovery continuum: target identification (genetic screening), target validation (genetically-modified cell lines), pre-clinical studies (genetically-defined in vivo models), and generation of improved biomanufacturing systems (genetically-modified CHO cells).

To allow scientists to study the effect of a genetic mutation in a variety of tissues, Horizon is now using CRISPR-Cas9 to engineer pluripotent stem cells (such as iPSCs). Critically these can then be differentiated into a variety of tissues types, such as liver or neural cells, all of which will have the same genetic background and the same introduced mutation. Researchers can then screen and validate compounds in these different but genetically identical backgrounds, making iPSCs a good model for pre-clinical studies (Figure 19).

Answer 1

Yes, the mention of CRISPR-Cas9 technology for engineering pluripotent stem cells to study the effect of a genetic mutation in various tissues indicates the presence of a single gene disorder being studied. This technology allows researchers to introduce specific mutations into cells, allowing for the study of the effects of that mutation on cellular function and potential therapeutic interventions.