Regulation of Self Renewal and Lineage Commitment in Human Embryonic Stem Cells

Funding Type: 
Comprehensive Grant
Grant Number: 
ICOC Funds Committed: 
Public Abstract: 

Human embryonic stem cells provide an indefinitely renewable source of any type of healthy human cell for use in research, or in transplantation therapy to treat a wide range of intractable, debilitating disorders characterized by cell loss or injury. To realize the promise of embryonic stem cell research, we must first learn to propagate stem cells on a large scale and to produce pure populations of desired cell types, for example cardiac muscle cells to treat heart disease. For some conditions we may require more than a billion cells to treat one patient. While this level of cell production is an attainable goal, at present our ability to grow stem cells on a large scale, or to obtain large numbers of specialized cells from them, is limited. This is because we do not fully understand the factors and conditions that provide for stem cell growth, or specialization into different types of body cells, and because under current conditions, stem cells can be unstable when grown in the laboratory. This research is aimed at achieving a better understanding of how stem cells multiply, and how they specialize to embark on the pathway to the formation of cells such as muscle, liver or brain cells. We will use new technical approaches to study the stem cell population in detail and to understand how to better control it. These studies also aim at ensuring that when we grow stem cells on a large scale in the laboratory, we avoid generating abnormal cells with genetic changes similar to those seen in cancer cells, to ensure safe use of stem cell derivatives in the clinic. The outcome of this work will be improved understanding of the molecular control of stem cells and improved technology for application of stem cells in research and medicine.

Statement of Benefit to California: 

Human embryonic stem cells can in principle provide an indefinitely renewable source of any type of normal tissue for use in research, including the development of new pharmaceuticals, and in regenerative therapy, to treat a wide range of debilitating disorders caused by cell injury or loss. The scope of the applications of human embryonic stem cell technology is enormous, and the major potential benefits to the economy and to public health that may follow from such research are widely recognised. The State of California is poised to take a leading role in this area internationally. In this field, as in other areas of research and development, platform technologies are a critical component of competitiveness. This project will accelerate the application of human embryonic stem cell technology by advancing our knowledge of how cultured stem cells grow and how they turn into specialized cell types. This basic knowledge will be essential to the development of methodology for large scale production of particular cell types from human embryonic stem cells. The Principal Investigator has experience in commercialization of stem cell technologies and our {REDACTED} at the {REDACTED} is well integrated into a medical school environment with excellent translational and clinical research capability. The Principal Investigator is also involved in a number of international initiatives and collaborations and his work will help to integrate stem cell research in California into these global efforts. Thus, we are well positioned to move the discoveries from our laboratory to the biotechnology sector, and into the clinic.