Molecular Regulation of Human Pluripotent Stem Cell Functions

Funding Type: 
Basic Biology III
Grant Number: 
ICOC Funds Committed: 
Public Abstract: 

Human pluripotent stem (hPS) cells can perpetually replicate themselves and can differentiate into virtually any type of cells in the adult body (pluripotency). Human embryonic stem (hES) cells, which are derived from blastocyst embryos, are the most studied type of hPS cells. The recent technological breakthrough has paved the way to convert adult differentiated cells such as skin cells into undifferentiated cells called human induced pluripotent stem (hiPS) cells that maintain pluripotency. Because of this enormous ability, hPS cells are thought to be the potential source of the future cell transplantation therapy for the treatment of diseases such as Parkinson's disease and diabetes mellitus.
Despite these promising functions of hPS cells, there are many biological questions and practical hurdles to overcome. Important issues include understanding how the unique functions of hPS cells are regulated and development of technologies to derive and grow hPS cells at high efficiency under completely animal-free conditions for future medical purposes.

We have recently identified a signaling pathway (like a hormone that mediates biological information) that controls cell-cell
communications and survival of PS cells. Moreover, by controlling this pathway, we have established a method to grow hPS cells under a fully animal-free condition.

In this proposal, we will investigate whether this signaling pathway also controls the unique functions of hPS cells.
Furthermore, by changing the activity of this signaling pathway, we will develop a new method to efficiently generate and expand new hiPS cell lines that are completely free from animal-derived materials.

Statement of Benefit to California: 

A major focus of our research proposal is to develop novel technologies by which human embryonic stem (hES) and human induced pluripotent stem (hiPS) cells can be propagated under completely defined conditions.
The establishment of such a new method would impact virtually all hES and hiPS cell-based application programs as it involves a
common basic process required to expand undifferentiated pluripotent stem cells before turning them into any type of adult cell for future therapeutic purposes.
It is therefore predictable that the new methodology will be promptly translated as an intellectual property to be commercialized, and would substantially activate the biotechnology field in the State of California. More importantly, the new methodology will be provided to the Institutes in California at the highest priority where the method will accelerate the process and continued research necessary to see the clinical application of pluripotent stem cell-based transplantation. Furthermore, the introduction of pluripotent stem cell therapies into California medical care as a direct result of our research and new methodology would further enhance the quality of medicine and medical technology available for California citizens.