The ability to modify the genes that are expressed in pluripotent stem cells or the mature cells produced from the stem cells will be important for many applications to regenerative medicine and for experimental research studies to understand better the biology of stem cells. For examples, inserting genes into stem cells may lead to cells with improved survival or ability to develop into specific mature cell types, may allow the developmental pathways to be studied and may be used to correct genetic diseases. Gene transfer into pluripotent stem cells is often limited by the transferred gene getting “turned off” as the stem cells grow into the desired type of adult cells. The primary goal of this project will be to develop improved gene delivery tools that will keep genes transferred into stem cells working and active. Studies will be performed to determine how the genes are turned off or kept on, to guide future development of optimal gene deliver tools. Diverse hESC and iPS lines will be studied to verify the generality of results; some of the hESC lines to be used cannot be studied with federal funds.
Statement of Benefit to California:
Development of methods for regenerative medicine using pluripotent stem cells will have wide-spread applications to improve the health and to provide novel, effective therapies for millions of Californians and tens of millions of people world-wide. Regenerative medicine may provide new treatments for diseases including diabetes mellitus, Parkinson’s disease, organ failure and injuries, inherited diseases and cancer and leukemia. The major challenge facing the field of regenerative medicine is to increase knowledge of the processes by which the mature cells of tissues (pancreas, brain, bone marrow, etc.) develop from stem cells, so that clinical approaches can be developed to produce cells suitable for transplantation. This Project will produce and apply novel tools for experimental studies of pluripotent stem cells and for the development of clinical therapies using hESC. The central focus is on the development of improved and optimal gene delivery vectors that can transfer and express new genes in stem cells, to influence their properties for research or for clinical purposes. These studies will help to advance the capacities for regenerative medicine. All scientific findings and biomedical materials produced from our studies will be publicly available to non-profit and academic organizations in California, and any intellectual property developed by this Project will be developed under the guidelines of CIRM to benefit the State of California.