Derivation of Customized Stem Cells for Regenerative Medical Therapy
Embryonic stem cells hold great promise in regenerative medicine for the treatment of numerous diseases, injuries, and disabilities. Despite recent clinical successes, there remain significant hurdles to establishing ethically sound, scientifically feasible, and practically realistic human stem cells that engender broad public support and exhibit convincing therapeutic effectiveness. Among these hurdles are the source (embryo vs adult) of stem cells and immune rejection following transplantation. To overcome these two hurdles, our long-term goal is to develop and apply efficient technologies for deriving pluripotent, embryonic-like stem cells from a patient's own tissues for the purpose of providing "customized", patient-specific regenerative therapy. The rationale behind our long-term goal is that the destruction of human embryos to derive new embryonic stem cell lines, and the clinical complications associated with rejection of transplanted stem cells that are not recognized as "self", prevent full realization of the enormous potential of regenerative therapies using stem cells.
Therefore, the overall objective of this SEED grant, which is the first step in achieving our long-term goal, is to extend and enhance existing technology for efficiently and reliably using adult human somatic cell nuclei to derive pure, pluripotent human embryonic-like stem cells. Our central hypothesis is that customized and therapeutically-useful embryonic-like stem cells can be derived from adult, human fibroblast nuclei reprogrammed in mouse oocytes. The justification for this project is that this methodology would eliminate the creation and/or destruction of human oocytes and embryos to derive patient-specific embryonic-like stem cells, preclude the need for immunosuppresvie therapy in patients receiving stem cells, and allow for the possibility of correcting inherited genetic mutations.
The research proposed here will significantly advance the field of stem cell biology, thereby promoting translational research applications that drive achievements of basic research to the patient's bedside faster and more effectively than before. By doing so, and by improving the health of the citizenry in need of regenerative medicine using stem cells, then this project will be of benefit to the State of California. In addition, this area of research will attract a broader and more diverse array of scientific experts in the field of stem cell biology to California, thereby also contributing to the advancement and development of the State's biomedical research enterprise.