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The Researchers

1) Photo: Ronald Worton
Ronald Worton
Understanding Muscular
Dystrophy

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Date and Place of  Birth:

Ronald Worton was born in Winnipeg, Manitoba on April 2, 1942.

Studies:

As a teenager, Worton's father encouraged him to pursue engineering, but when he attended the University of Manitoba, his interests shifted to mathematics and physics. With a desire to pursue a discipline with more practical applications and a better opportunity for social interaction, pure theoretical physics lost its appeal and Worton switched into Biophysics - the scientific study of the physics behind the biological structure and processes of organisms. In 1969, he received a Ph.D. in Medical Biophysics from the University of Toronto and spent two years as a Postdoctoral Fellow in Biochemistry and Biophysics at Yale University.

Mission:

In 1971, Worton joined the Department of Genetics at the Hospital for Sick Children (HSC) in Toronto, at a time when the scientific community was just beginning to recognize that a number of diseases were caused by genetic defects. Worton realized that to facilitate a better understanding of human disease, one would have to develop an appreciation for what genes are, how they function and the consequences of genetic defects. Thus, he began a new research program to identify the gene that is defective in children with Duchenne Muscular Dystrophy (DMD), a disease characterized by the degeneration of muscle fibers, and for the next several years Worton concentrated his research efforts on this extensive project.

Achievements:

In 1986, Worton's hard work paid off when his research group identified the gene encoding dystrophin (a very large protein that stabilizes the muscle fibres when they contract and expand) and the genetic alterations that are responsible for DMD. This concerted effort was not only a breakthrough discovery, but also a prototype gene discovery that provided scientists with a set of procedures to follow when studying the genetic defects underlying human disease.

 

The discovery of the gene for dystrophin offered both good and bad news; good news because it was finally understood that the lack of dystrophin inmuscle fibres was the cause of the muscle degeneration, and bad news because restoring dystrophin to the muscle would be a tremendous task, involving the replacement of the defective gene with a good gene, in order for the muscle fibres to manufacture the missing protein. Worton dabbled in gene therapy technology for a while but eventually decided that gene therapy would only prevent further degeneration of muscle fibres, not replace the damaged muscle.

 

Having concluded that only stem cells would have the power to repair the damaged muscle he prepared several successful proposals to federal and provincial governments to build, equip and staff a new Centre for Stem Cell and Gene Therapy at the Ottawa Health Research Institute. In addition he knew many talented stem cell researchers across Canada and led them in a proposal to the federal government to create a Network of Centres of Excellence dedicated to stem cell research.

In April 2001, he successfully founded and became the Scientific Director of the Stem Cell Network, with a mandate to bring together basic scientists, clinical scientists, engineers and social scientists "to explore the social, ethical, legal and policy issues inherent in stem cell research and therapeutics, as well as develop new therapies for chronic diseases."

Worton moved to Ottawa in 1996 with the challenge of building a new hospital based Research Institute. Following a series of Hospital and Institute mergers, Worton is now CEO and Scientific Director of the new Ottawa Health Research Institute, and Vice President of Research at the Ottawa Hospital. His mandate is "to build an Institute that has outstanding scientists who will discover the cause, cure and prevention for a host of diseases including, he hopes, DMD, Diabetes, Stroke and Obesity." Always looking toward the future, Worton is making sure the Institute moves forward and capitalizes on opportunities and new technologies to remain at the leading edge of research. He predicts that stem cell research will flourish over the next five years and that within 10 years, medicine will be regularly using stem cells for the treatment of a variety of genetic diseases that are currently untreatable.

Worton credits his relationship with Louis Siminovitch, former Head of the Department of Genetics at Hospital for Sick Children, as having a profound influence on his career, and for being a wonderful mentor, advisor and friend.

Favourite Food:
All.

Favourite Music:
Classical and music from the 40's and 50's.

Favourite Book:
Books regarding the history of science.

Favourite Movie:
Shrek.

What inspired me as a teenager?
Hockey.

Favourite Subject in High School:
Mathematics and Physics.

Favourite Hobbies:
Watersports like boating, swimming and waterskiing.

Credo:
"You have an obligation to do something with your life that will make a difference."

 

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Image credits: 1) Ronald Worton.