Pluripotent stem cells have enormous potential for developing new treatments for a myriad of human diseases and disorders. In addition they provide a unique window into the development of our own species. In order for these stem cells to reach their full potential we must have a much better understanding of how they grow and how they can be turned into specialized cells that could be used to treat patients. The present proposal describes studies designed to study a new factor that seems to be important for the growth of stem cells. This factor is part of a complex of proteins whose function in stem cell biology had not been appreciated. Understanding how this protein complex works in stem cells could aid in our development of new cell-based therapies and give new insights into the mechanisms that normally control human development. In addition understanding how these proteins work could aid in the development of new stem cell lines from patients which could then be used to study disease processes.
Statement of Benefit to California:
A goal of Prop 71 is to turn stem cell research to clinical application. Disability and loss of earning resulting from a disease or disorder are devastating and are a financial burden for California in addition to patient suffering. Therapies using human ES cells and induced pluripotent stem cells (hiPSCs) could change lives. Using these cells as models of disease will help us understand underlying causes and aid drug development. For this potential to be realized, we need a better understanding of how stem cells grow and what regulates their self-renewal. Maintenance of stem cells in an undifferentiated state is problematical and long term growth of stem cells can be associated with genetic alterations, some associated with cancer. Understanding mechanisms regulating stem cell growth will be important in maintaining stem cells, understanding both how to differentiate them to specialized cells and the risks of transplanting cells into patients. The proposed research should provide fundamentally better understanding of how hESCs and hiPSCs grow and self-renew. Anticipated benefits of our research to Citizens of California include: 1. Development of improved methods for growing stem cells and developing cell-based treatments 2. Understanding risks of transplantation 4. Improved understanding of early embryo development 6. Revenues from technology and intellectual property 7. Creation of new biotech companies 8.Creation of new jobs in the biotech sector.