The man who created a mouse

Dr. Markus Grompe

Dr. Markus Grompe selected as winner of 2018 Richard Jones Distinguished Alumnus Scientist Award

May 14, 2018

Story by Todd Murphy, photo by David Wakeling

Alongside everything Markus Grompe, M.D. R '87, has accomplished, there is this: He is the man who created a mouse.

Dr. Grompe is an international pioneer in stem cell and gene therapy research. He identified and cloned a key gene linked to the rare genetic disease Fanconi anemia. And he's made, and continues to make, major discoveries in the treatment of liver disease. Recently, he and his team have begun focusing on using gene therapy one day to cure diabetes.

But it's the genetically engineered mouse that he semi-accidentally created 25 years ago that he might be most known for. He created the transgenic mouse to study a rare disease called tyrosinemia that can cause kidney and liver dysfunction. But the altered mouse ended up being much more valuable: it has a "humanized" liver, which has allowed scientists around the world to use it to study all sorts of metabolic functions, including how drugs are metabolized by humans.

"This is literally what my career is built on – this knockout mouse," Dr. Grompe said, proudly showing a photo of himself 24 years ago with one of the first mice.

Dr. Grompe became the first director of the Oregon Stem Cell Center at OHSU in 2004. In 2008, he became director of the Papé Family Pediatric Research Institute at OHSU Doernbecher Children's Hospital. But he's always been more than a researcher.

Because of his work, he's an expert in a range of diseases called "inborn errors of metabolism" – which often come from a defect in a single gene. He uses that knowledge as a Doernbecher physician, helping patients and families who come from throughout Oregon and nearby states for his expertise.

"I would never give that up," he said. "It's really part of who I am – that direct interface with patients."

It's also exhilarating, he said, to sometimes tell patients that there's now a treatment for a condition no one had been able to help them with. "I've seen many, many diseases that were untreatable when I started," Dr. Grompe said. "And we now have a therapy for them. Being able to do that for a patient is really satisfying."

Dr. Grompe is Ray Hickey Chair of the of Pape' Family Pediatric Research Institute and professor of pediatrics and molecular and medical genetics, OHSU School of Medicine.