Generation of pluripotent stem cell lines to test novel compounds for the treatment of disease

Summary

iPSCs are somatic cells (non-reproductive cells) that are manually reprogrammed in the laboratory to become pluripotent, that is they have an ability to become different cell types.

With this study, we plan to generate an induced pluripotent stem cell (iPSC) line from each donor.  The iPSCs can then be used as a research tool in studies investigating the disease, drug discovery and pre-clinical evaluation of potential new treatments. 

This reprogramming technology offers several advantages for researchers which include:

• the ability to generate several different cell types from one iPSC culture,
• the potential to study many different diseases (common and rare) and their treatment,
• tissue regeneration capabilities,
• and it is an ethically non-controversial strategy to generate patient-specific stem cell lines.

This research will further our understanding of the biology of the retinal disease and may lead to the development of potential treatments for the major causes of blindness.