Research Advisory Council

Jim and Ann Orr Massachusetts General Hospital Research Scholar, J. Keith Joung, MD, PhD

Engineering Genes to Understand and Treat Disease

J. Keith Joung, MD, PhD, likes to tinker with things to get them to work. Deep down, he’s an engineer at heart. His challenge is to figure out how — at the cellular level — researchers can engineer genes to improve the study and treatment of disease.

Keith Joung, MD, PhD, 2011 MGH Research Scholars

Jim and Ann Orr Research Scholar,
J. Keith Joung, MD

Dr. Joung went to medical school because he wanted to help patients. Along the way, he learned that through research he could find cures for disease. Today, he focuses his research on technologies that can be used to treat a wide variety of diseases. Fortunately, Dr. Joung is in the best place to do just that.

Massachusetts General Hospital has the largest hospital-based biomedical research program in the United States and attracts many of the world’s brightest medical researchers. But in today’s climate of tight funding, mid-career scientists, like Dr. Joung, find themselves faced with a dilemma. They cannot pursue projects with unknown outcomes that promise bold advances simply because many funders want to invest in ideas that are easier to prove.

With support from the Jim and Ann Orr MGH Research Scholar Award, Dr. Joung has been given the freedom to chase the big ideas. The MGH Research Scholars Program is helping scientists perform research that challenges the field to think about disease in new ways. One of the first couples to step forward to support the scholars program, Jim and Ann Orr, donated $500,000. Mr. Orr says he hopes his family’s gift will encourage others to support these promising scientists.

He understands great science requires creative exploration. “A series of questions gets asked, and another set of questions gets asked that takes the research down a different path, and, I think, that’s very exciting,” Mr. Orr says.

Engineering Tools with the Goal of Curing Disease

Dr. Joung works with zinc-finger nucleases, which are man-made proteins that have two functions. First, zinc-fingers act like keys. Just as the notches in the key to your front door exactly fit that lock, researchers, like Dr. Joung, are trying to engineer zinc-fingers to recognize particular DNA sequences within genes that cause disease. The better the key fits, the better the potential outcome for patients.

Secondly, the nuclease, an enzyme attached to the zinc-finger, acts like a pair of scissors. The nuclease cuts DNA, which allows researchers to use the body’s normal repair processes to correct mutations in genes that cause disease. Dr. Joung’s lab is developing zinc-finger nucleases that might be used to study and treat diseases like cystic fibrosis and sickle cell anemia.

The promise of Dr. Joung’s work goes beyond any one disease. His lab has spent the past few years developing methods and making materials publicly available to academic researchers who want to create their own zinc-fingers to further their research.

At Mass General there are many possibilities for collaboration, due to the breadth of research carried out here. Dr. Joung has an ongoing collaboration with Randall T. Peterson, PhD, the Charles and Ann Sanders MGH Research Scholar, who is using zinc-fingers in zebrafish to model human diseases, including schizophrenia and Alzheimer’s disease.

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For information about how you can support the Research Scholars Program, please contact the Mass General Development Office at 617-726-2200

A Critical Time to Support the Best of the Best

Dr. Joung began his work with zinc-finger nucleases in 1998. He says the field today is more exciting than ever. He plans to use the Jim and Ann Orr MGH Research Scholar Award to expand his research and develop new tools to introduce mutations into genes, and, of course, continue to improve zinc-fingers.

“I am very appreciative that Jim and Ann Orr have the foresight to fund high-risk transformative research and trust that MGH and we will use the money well,” Dr. Joung says.

Competition for the awards is intense. During the program’s inaugural year, more than 115 researchers applied. Five researchers, the best of the best, including Dr. Joung, who is associate chief for Research in Mass General Pathology, were named MGH Research Scholars.

“Dr. Joung’s work, particularly his efforts to make information on how to create zinc-finger nucleases publicly available, has helped many scientists. He understands the scientific community is larger than any single person or research group, and, together, we can answer the major questions of the day,” says Bruce Walker, MD, acclaimed physician-researcher and co-chair of Mass General’s Research Scholars Award Committee.

For information about how you can support the Research Scholars Program, please contact the Mass General Development Office at 617-726-2200.