Regenerative medicine using human pluripotent stem cells (hPSCs) holds a great promise in developing therapies for treating developmental abnormalities, degenerative disorders and aging-related illness. However, today’s hPSC collection faces two major challenges: shortage in supply and uncertain safety. Recently, the development of human induced pluripotent stem cells (hiPSCs) has solved the shortage problem by manually reprogramming resourceful somatic cells to hiPSCs. Scientists have been using these hiPSCs to conduct research and develop cures on multiple fronts, including testing drugs, developing therapies, devising tests for earlier diagnoses, and generating tissues/organs for transplantation. To further advance these research fronts, we need pure hiPSCs to prevent uncertain safety. As multiple factor-induced hiPSCs often present cell heterogeneity that leads to uncertain safety, this proposed project adopts a single factor – miR-302 induction method for hiPSC derivation. MiR-302 is a small regulatory microRNA that can replace all previously defined reprogramming factors for inducing homogeneous hiPSC formation. This microRNA-induced hiPSC technology is developed in California and has a higher reprogramming efficiency than previous Yamanaka’s methods using multiple factors. With these advantages, the hiPSCs provided by this project can serve as a better resource to the research and drug development community for advancing the progress of modern regenerative medicine.
Statement of Benefit to California:
The goal of CIRM is to establish a solid foundation for developing the future regenerative medicine industry in California. In order to achieve this goal, California needs its own pluripotent stem cell resource for further development. However, the legal right of using human embryonic stem cells (hESCs) belongs to WiCell Research Institute in Wisconsin, USA. Alternatively, OSKM-induced hiPSCs may replace hESCs but this technology is owned by Kyoto University in Japan. To solve this issue, PD has devised a new miR-302-induced hiPSC technology in California and is willing to contribute his right and efforts to achieve the same goal of CIRM. With this novel technology, California can freely develop its own regenerative medicine technologies to compete with other leading groups in the world. Since miR-302 also functions as a tumor suppressor in humans, the hiPSCs generated by PD’s method has an advantage in safety and hence may have a better chance to pass FDA approval. In light of these advantages, California has actually possessed all expertise, technologies, legal rights and resources required for the development of a top notch regenerative medicine industry in the world. To achieve this goal, we just need to coordinate these experts, technologies, legal rights and resources into one collaborative system. As the regenerative medicine market is expected to reach $1.4 billion dollars in 2012, a fruitful reward of this California-based system is highly foreseeable.