Shoukhrat Mitalipov, Ph.D., principal investigator for the Center for Embryonic Cell and Gene Therapy. (OHSU/Kristyna Wentz-Graff)

Biography

Shoukhrat Mitalipov, Ph.D., directs the OHSU Center for Embryonic Cell and Gene Therapy, where he is focused on developing treatments to prevent the transmission of genetic disease from parent to child. Mitalipov resides in Beaverton, Oregon, and is a professor at the Oregon National Primate Research Center with appointments to the departments of Biomedical Engineering; Obstetrics and Gynecology; Pediatrics; and the Knight Cardiovascular Institute in the OHSU School of Medicine.

Mitalipov was born Aug. 19, 1961, in Almaty, Kazakhstan. He earned his Ph.D. in 1994 from the Research Center for Medical Genetics in Moscow. He arrived at OHSU in 1998 after conducting postdoctoral research in stem cell and developmental biology at Utah State University.

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Discoveries

This sequence of images shows the development of embryos after co-injection of a gene-correcting enzyme and sperm from a donor with a genetic mutation known to cause hypertrophic cardiomyopathy. A new study, published today in the journal Nature, demonstrates an effective method of repairing a disease-causing mutation from the moment of fertilization, preventing it from being passed to future generations. (OHSU)

In August 2017, Mitalipov and colleagues published groundbreaking research in Nature demonstrating that the gene-editing tool CRISPR can be effectively used to repair a disease-causing mutation in a human embryo and prevent the disease from being passed down to future generations. The focus of the study was the gene mutation that causes hypertrophic cardiomyopathy, a common inherited condition that can cause sudden cardiac death and heart failure. This is the first time scientists have successfully tested the gene repair method on donated clinical-quality human eggs. Results of this landmark research, verified in 2018, were reported in thousands of media outlets worldwide.

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In 2013, Dr. Mitalipov and his colleagues developed a new process to create human embryonic stem cells from a patient’s skin cells, a discovery that could lead to treatments for a vast range of conditions caused by diseased or injured cells. The discovery was named among the top 10 scientific breakthroughs of the year by Nature, Science, Time, Discover and National Geographic. 

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Mitalipov also is renowned for developing a technique to break the cycle of diseases passed from mother to baby through mutations in mitochondrial DNA. Mitochondria control respiration and energy production within every cell of the body, so mutations in mitochondrial DNA can cause a range of potentially fatal disorders affecting organs with high-energy demands such as the heart, muscle and brain. Mitalipov's mitochondrial replacement technique mitigates this problem by replacing mutant mitochondria in a mother's egg with donor mitochondria. He successfully demonstrated this technique with rhesus macaques in 2009 and in human cells in 2012. Clinical trials are already permitted in the United Kingdom. In 2015 he published a breakthrough in Nature that set the stage for replacing diseased tissues in patients with mitochondrial disease.

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Mitalipov holds seven U.S. patents in techniques involved in gene therapy approaches for both nuclear DNA and mitochondrial DNA.