This research project is aimed at developing a new form of liver transplantation to treat birth defects that prevent normal liver function using liver cells isolated from discarded fetal tissues. It is now possible to detect many inherited diseases early in pregnancy. It may also be possible to treat diseases that prevent normal stem cell function by transplantation of healthy stem cells before birth. There are potential advantages to such a therapy such as early treatment of disease and a lower risk of rejection because the fetal immune system is not developed. This proposal studies a number of methods aimed at testing the effectiveness of transplanting liver stem cells into a fetus to treat liver disease. Often transplanting healthy cells offers treatment or a cure for many genetic diseases as well as other illnesses caused by viral infection or cancer. However, a lack of available or suitable donor tissue prevents such therapy in many cases. Fetal tissues are a source of stem cells available from elective abortions that are generally discarded. Harvesting liver cells from these tissues to treat patients with liver diseases offers the possibility of increasing the pool of donor cells that are available transplantation. This proposal studies the feasibility of banking fetal liver for transplantation.
Statement of Benefit to California:
Liver diseases caused by viral infection, drugs or inherited disease affect many thousands of Californians. Often, transplanting healthy cells offers treatment or a cure for many of these diseases, but a lack of available or suitable donor tissue prevents such therapy in many cases. Use of fetal stem cells offers the hope of generating a greater supply of tissues for cellular therapy. The development of prenatal liver transplantation would also offer additional means to treat birth defects that affect normal liver function. The successful outcome of this work will offer new hope to many Californians suffering from liver diseases. This will improve lives and save money on long-term health care costs associated with these diseases. Development of the technologies and expertise to bring these novel forms of therapy from the laboratory bench to hospital bedside will also keep California in the forefront of the biotechnology industry, will attract talented scientists and clinicians to California and will create high-paying jobs.