OHSU researchers successfully test new gene therapy in human cells

Imagine knowing that your child is at risk for inheriting a genetic condition. Now imagine being able to fix the genetic defect before the child is even born. This may sound like something out of science fiction, but we’re one step closer to it being reality thanks to researchers here at Oregon Health & Science University.

Shoukhrat Mitalipov, Ph.D., associate scientist in the Division of Reproductive & Developmental Sciences at the Oregon National Primate Research Center, and Paula Amato, M.D., associate professor of obstetrics and gynecology in the OHSU Center for Women’s Health, have collaborated to test a new gene therapy method of preventing inherited disease in human cells for the first time. The results of their study will be published in Nature.

Here’s how it works: Mitochondria in reproductive cells contain genetic material just like the cell nucleus. In 2009, Dr. Mitalipov developed a method to extract this damaged DNA and replace it with healthy donor mitochondrial genes. A major upside to this method is that the cells retain 99 percent of their original DNA–only the damaged genes in the mitochondria are removed. Dr. Mitalipov tested this method in a nonhuman primate model back in 2009, but this is the first time the procedure has been successfully performed in human cells.

This research was supported by grants from the OHSU Center for Women’s Health Circle of Giving and additional private funds from OHSU as well as the Leducq Foundation and the OHSU Vice President for Research office. An additional nonhuman primate study to demonstrate that this method works in frozen and thawed monkey embryos was supported by grants from the National Institutes of Health.

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