Question

is there any single gene disorder mentioned in here. please mentiion what single gene disorder have been mentioned in this peice of evidence

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

[AI-generated answer — Bot GPT 3.5]

In this piece of evidence, the single gene disorder mentioned is not specified. However, the use of CRISPR technology by Horizon Discovery to engineer pluripotent stem cells for studying the effect of genetic mutations in different tissues could potentially be used to model single gene disorders such as cystic fibrosis, sickle cell anemia, or Duchenne muscular dystrophy.