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New Gene Therapy Breakthrough in Humans is Result of Research Pioneered at OHSU's Primate Center

A promising gene therapy method first developed through animal studies at Oregon Health & Science University has now been successfully tested using human cells. Today, the scientific journal Nature published results of a study conducted at Newcastle University in the United Kingdom where a nucleus from one embryo was transferred to another.

For more information about the breakthrough visit

It is believed that by using this method of gene transfer, genetic diseases carried in cell mitochondria might be prevented, and maternally-based diseases that are frequently passed from generation to generation could be halted.

Today’s breakthrough using human cells was previously accomplished in rhesus monkey cells by Shoukhrat Mitalipov, Ph.D., and colleagues at the OHSU Oregon National Primate Research Center. Nature published the Oregon-based breakthrough in August 2009. The research resulted in the birth of healthy twin monkeys nicknamed “Mito” and “Tracker” (in reference to the procedure used for mitochondria imaging). These monkeys are the world’s first animals derived by mitochondrial gene transfer. At the time, the OHSU research breakthrough was covered by news outlets worldwide.

“I congratulate the Newcastle research team for their success,” said Mitalipov, whose laboratory pioneered method for marking DNA contained in the egg nucleus so that it could be transferred from one egg to another. Because DNA was not previously visible to the naked eye, this was a significant barrier to successful DNA transfer.

“This is another important step in preventing mitochondrial-based diseases, which include certain forms of cancer, diabetes, infertility and neurodegenerative diseases,” added Mitalipov. “However there is still much more work to be done. Additional research and safety studies must take place before the research progresses further in humans. In addition, extensive ethical considerations must take place to ensure that this technology is used in an appropriate fashion.”  

Summary of OHSU’s previous research
Scientists collected groups of unfertilized eggs from two female rhesus macaque monkeys (monkeys A and B). They then removed the chromosomes, which contain the genes found in the cell nucleus, from the eggs of monkey B, and then transplanted the nuclear genes from the eggs of monkey A into the eggs of monkey B. Then the eggs from monkey B, which now contained their own mitochondria but monkey A’s nuclear genes, were fertilized. The fertilized eggs developed into embryos that were implanted in surrogate monkeys. The initial implantation of two embryos resulted in the birth of “Mito” and “Tracker.”

More information about OHSU’s 2009 breakthrough.

The ONPRC is a registered research institution, inspected regularly by the United States Department of Agriculture. It operates in compliance with the Animal Welfare Act and has an assurance of regulatory compliance on file with the National Institutes of Health. The ONPRC also participates in the voluntary accreditation program overseen by the Association for Assessment and Accreditation of Laboratory Animal Care International (AAALAC).

About OHSU   
Oregon Health & Science University is the state’s only health and research university, and Oregon’s only academic health center. OHSU is Portland's largest employer and the fourth largest in Oregon (excluding government). OHSU's size contributes to its ability to provide many services and community support activities not found anywhere else in the state. It serves patients from every corner of the state, and is a conduit for learning for more than 3,400 students and trainees. OHSU is the source of more than 200 community outreach programs that bring health and education services to every county in the state.

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