Center for Embryonic Cell and Gene Therapy

Shoukhrat Mitalipov, Ph.D.

The Center for Embryonic Cell and Gene Therapy, led by Shoukhrat Mitalipov, Ph.D., is the 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.

Why now?

There has never been a more exciting time in cell and gene therapeutics research. There has also never been a more urgent need for health advocates to advance this field through leadership philanthropy. Even established programs that are truly breaking new ground face challenges securing federal grants. Support from private foundations and individuals will be the catalyst that helps this work deliver on its exceptional promise to prevent disease and promote human health worldwide. OHSU presents an exceptional opportunity to invest in cure-focused translational research under way at the laboratory that is now revolutionizing this dynamic field.


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

Another facet of Dr. Mitalipov's work could, for the first time in history, break the cycle of diseases passed from mother to baby through mutations in mitochondrial DNA. Such mutations impact one out of every 4,000 babies born, causing a wide variety of diseases including autism, blindness, muscular dystrophy, cardiovascular disease, and seizures.

Dr. Mitalipov's mastery of cell manipulation is at the heart of a promising therapy that could prevent inherited mitochondrial diseases. The method, 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.