The Center for Embryonic Cell and Gene Therapy at Oregon Health & Science University, led by Shoukhrat Mitalipov, Ph.D., is pioneer of the technique to produce rejection-proof, genetically matched embryonic human stem cells from skin cells using somatic cell nuclear transfer (SCNT). Our program connects basic cell science, translational research and clinical testing in a virtuous cycle of discovery and innovation. Research performed at the center will keep people healthier as they age, restore mobility for patients with Parkinson's disease or spinal injuries, and stop familial diseases before they affect a new generation.
A groundbreaking discovery
In May 2013, Dr. Mitalipov and his team published a study in Cell that describes a new process for creating human stem cells from skin cells. Stem cells are thought to hold promise for treating various degenerative diseases, but finding a source of embryonic stem cells—which can be reprogrammed into any other cell type—has been an obstacle to progress in developing such treatments. The process described by Dr. Mitalipov, referred to as SCNT, removes chromosomes from a human donor's unfertilized egg and then replaces them with new DNA from a patient's skin cells. This discovery sparked more than 2,000 news reports internationally and was named a top 10 scientific breakthrough of 2013.
SCNT provides an alternative to stem cells derived from fertilized human embryos. It may also provide advantages over the induced pluripotent stem cell (iPSC) method, which involves genetic programming changes in mature skin cells to transform them to an embryonic-like state.
Breaking the cycle of disease
Dr. Mitalipov's work could, for the first time in history, break the cycle of disease passed from one generation to the next using germline gene therapy approaches for both nuclear DNA and mitochondrial DNA. Such mutations impact a great number of families every year, causing a wide variety of diseases. Dr. Mitalipov's mastery of cell manipulation is at the heart of promising therapies that could prevent inherited diseases.
Mitochondrial replacement therapy, first proven in non-human primate models in 2009, was demonstrated successfully in human cells in 2012. It involves replacing a mother's mutated mitochondrial DNA with the equivalent material from a healthy egg, effectively retaining 99 percent of the mother's DNA but with healthy mitochondrial DNA. Upon FDA approval, this technique is ready for human clinical testing in the United States. Inherited genetic disorders affect millions of people worldwide, using current technologies, Dr. Mitalipov's most recent work is exploring the safety and efficacy of human germline gene correction.
By giving much needed resources to the most creative minds in biomedical research you will accelerate scientific discovery and development of novel therapeutic approaches involving stem cells and gene therapy. Much of our lab's success can be attributed to gifts from individual donors who supported our research projects not yet eligible for Federal funding.
Philanthropic gifts from corporations and foundations provide critical money for our new research initiatives on human embryo and stem cell research. These partnerships will pave the way for some of the most exciting and fruitful advances in biomedical research - from using stem cells in regenerative medicine to novel gene therapy approaches for the treatment of inherited human conditions.