Engineering, economical or biological sciences have undeniably contributed to an increase in the standards and quality of life: we live longer and mostly better. However, medicine has yet to bring cures for many major diseases, such as neuronal degenerative diseases, which might touch every second elderly person above the age of 80, heart diseases, which are the leading cause of death in developed countries, diabetes, cancers, and so many more. Human embryonic stem cells, which are obtained from oocytes of supernumerary embryos, appear as a major contributor of innovative therapies for those diseases, whereby they could replace the sick cells, or deliver necessary factors. But they will not be used as such, mostly due to the ethical issues they raise, and because of the difficulty in obtaining them.
A great breakthrough in stem cell research allows for imagining that the therapeutic potential of those cells could be carried out by reprogrammed adult cells in the near future. Cells have indeed been obtained from adult skin cells, using some genetic reprogramming, that can give rise to any kind of cell in the human body. This is only the first of many steps to be taken in order to achieve available cures for the above mentioned diseases.
An important issue, which will need to be solved, is the safety of the reprogramming method. As it stands now, cells are permanently genetically modified, and are very likely to give rise to cancers. This, of course, is not viable for therapeutic use. Moreover the extent of similarity or dissimilarity of the generated cells with human embryonic stem cells and with cells that could be used in a clinical setting has yet to be fully characterized, and the mechanism by which reprogrammed cells are able to give rise to any cell type is very poorly understood.
This research project elaborates a strategy to obtain, in a safer way, those invaluable cells, and plans to make sure that the obtained cells would be usable for therapeutic applications. In order to do so, we will concentrate in reprogramming human spermatogonial stem cells, which can be obtained by a simple biopsy, and which are not as differentiated as the skin cells used previously, which should make reprogramming easier. We will study the mechanism of reprogramming, which is up to now still a big black box, and hope thereby to improve the efficiency of any type of reprogramming. We will generate pluripotent stem cell lines, which can be later used for both basic and applied research.
Statement of Benefit to California:
The people and the state of California can gain both scientifically and economically from the work proposed here. Scientifically, California is leading the way in stem cell research and will benefit both from the fruits of our research, and from the added-value to individual scientific efforts conducted in California brought by the establishment of the network of collaborations, both within the State, and around the world, which we will put into place. We conduct research to expand fundamental knowledge of human biology and to bring science close to patients’ bedsides. In this study we would develop an approach that allows the production of unlimited numbers of different pluripotent cell lines, which could later be used for therapeutic purposes, or as disease models by the pharmaceutical industry in order to test new drugs. In the future, those cells may hold the key to replacing cells lost in many devastating diseases such as Parkinson’s and diabetes. Moreover, as the pluripotent stem cell lines developed in our laboratory will be made available to all academic researchers around the world, we are convinced that our technology will have wide-spread use by stem cell researchers and geneticists in California as well as the rest of the world. Economically, the technologies developed or improved throughout our proposed research could lead to economic growth through the creation of new products, technologies, jobs, companies and even new industries and most importantly to advances in health care and improvements in the quality of life.