Human embryonic stem cells (hESCs) have been made and kept alive using many different methods. Researchers throughout the world have noticed that different stem cells have different qualities, but no-one has conducted a systematic comparison of multiple hESCs that have been made or kept alive using different methods. In this study, we will generate and compare hESCs using 4 different methods. 3 of these methods will result in the generation of patient-specific hESC lines, which would not be rejected if ever administered to a patient as a treatment. All of the methods will use techniques that do not involve rodent reagents and are FDA-compliant, such that all of the resulting products will be clinical grade. In addition, to generate some of our new lines, we will use starting material from genetically-diseased individuals so as to generate cellular models of human disease. Such disease lines will be very useful to study disease, and generate drugs that may counteract disease progression. Our systematic characterization and comparison of various lines that we generate will allow us to better define the appropriate uses for each line in research and therapy. Our laboratory is uniquely positioned to attain these goals, given our lengthy experience with hESCs, our experience in making new hESC lines, our published differentiation protocols, our IRB approval for all proposed studies, and our uniquely large supply of starting material from which to make the lines.
The proposed research will benefit California by preserving and strengthening the State’s position as a leader in the field of stem cell tools and therapeutics. Through the passage of Proposition 71 and subsequent establishment of the California Institute for Regenerative Medicine, the voters of California have identified stem cell research as a key area of focus for the State, with anticipated positive impacts including the creation of biotechnology jobs, attraction of leading researchers to California universities, creation of valuable intellectual property, and advancement of therapeutics beneficial to California residents. The studies outlined in this proposal will lead to the development of new technologies that will form valuable intellectual property. Commercial interest in these technologies could then lead to biotechnology jobs. If successful in the generation of patient-specific stem cell lines, these studies will make a significant contribution to the development of patient therapies. Given the number of diseases that have the potential to be addressed with patient-specific cell lines, the emotional and financial benefit to Californians could be considerable.