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A team effort: Institute for Translational Neuroscience, key collaborations for accelerating ataxia research take shape

When it comes to finding a cure for ataxia, most researchers would agree it takes a village—a community of dedicated investigators from various disciplines and specialties who together can take findings from Petri dishes and turn them into valuable treatments for patients.

Ataxia researcher Harry Orr, Ph.D., says partnerships through the new Institute for Translational Neuroscience will help bring researchers closer to finding a treatment for ataxia.

At the University of Minnesota, interdisciplinary research teams are continually working toward that goal. Now, the newly formed Institute for Translational Neuroscience (ITN) is providing a venue to make a good thing even better.

In July, the University agreed to provide $1.8 million annually for research within the institute, which includes an area focused on neurodegenerative and neuromuscular diseases. The ITN brings together the expertise of researchers in disease areas such as ataxia, muscular dystrophy, Parkinson’s disease, Huntington’s disease, amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease), and other related disorders.

“The ITN aims to enhance neuroscience research at the University as it relates to neurological disorders, which runs the gamut from understanding the basic mechanisms of these conditions all the way to creating new treatments for them,”says Harry Orr, Ph.D., a lead ataxia researcher and director of the ITN. “The stronger we are in this area generally, the better we will be positioned in terms of ataxia specifically.”

University investigators are enthusiastic about having the infrastructure in place to foster further collaborations and thoughtful dialogue about research questions.

“The institute brings together very basic neuroscientists with molecular biologists with clinicians, allowing us to model in mice and even flies diseases seen in the clinic,” explains Laura Ranum, Ph.D., an ataxia researcher and research director of the Wellstone Muscular Dystrophy Center. “My hope is that we will be able to translate what we find in the laboratory into successful approaches for treating ataxia and other diseases back in the clinic.”

Already, Ranum has seen the benefits of the University’s collaborative culture. By combining their expertise, she and colleague John Day, M.D., Ph.D., have identified both ataxia and muscular dystrophy genes. Ranum’s lab has used these discoveries to develop mouse models that mimic the human diseases spinocerebellar ataxia type 8 (SCA-8) and myotonic dystrophy type 2, the most common form of muscular dystrophy in adults.

“In the process, we have learned that mutations that cause myotonic dystrophy have striking similarities to those that cause SCA-8,” Ranum says. “From a biological viewpoint, these diseases are highly related to each other.”

John Day, M.D., Ph.D. and Laura Ranum, Ph.D.

Through the ITN, Ranum will now be working with Department of Neuroscience chair Timothy Ebner, M.D., Ph.D. Ebner has refined functional imaging techniques that will give researchers clues about how neurons work in the brain. By taking images of the brains of Ranum’s mouse models, Ebner is shedding light on how neuron signaling goes awry in people with myotonic dystrophy and SCA-8.

Orr, too, is looking forward to seeing how the ITN will benefit ataxia research. He hopes to recruit 18 new translational research scientists over the next several years to hasten progress toward new treatments.

“We have evidence that many of the processes that are affected as neurons or muscle cells become dysfunctional are common among the various neurodegenerative and neuromuscular disorders, so for each of the different diseases, researchers are asking the same questions,” says Orr. Finding answers for Parkinson’s disease or muscular dystrophy, he explains, will also help advance scientific knowledge about ataxia.

“I want to find a treatment for ataxia in the next 10 years,” he says. “Given that goal, and with partnerships through the ITN, I can’t think of a better place to be.”

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