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September 12, 2005
Yerkes to create first transgenic model for Huntington's

BY Stephanie McNicoll

In the first study of its kind, researchers at the Yerkes National Primate Research Center will develop a transgenic nonhuman primate model for inherited, neurodegenerative diseases. The goal of the four-year, National Institutes of Health (NIH)-sponsored study is to develop treatment and prevention options specifically for Huntington’s disease.

Transgenic animals are those who have genetic information from another species inserted into their DNA. Researchers have used transgenic mouse models for Huntington’s disease to identify the genetic defect that causes the disorder.

Anthony Chan, assistant professor of human genetics, and his research team will use transgenic nonhuman primate models to study the onset and progression of Huntington’s, a genetic, neurodegenerative disorder that causes uncontrolled movements, loss of mental processing capabilities and emotional disturbance. They also will compare neurological changes in the transgenic model with the neurology of human Huntington’s patients. The pioneering study serves as an example of the critical role nonhuman primates play in better understanding human disease.

“Neurodegenerative diseases like Huntington’s disease are complex disorders that involve genetic defects and physiological changes, including the death of neurons that lead to a wasting away of the brain,” Chan said. “Because of their genetic and behavioral similarities with humans, transgenic nonhuman primate models will allow us to clarify the correlation between defects and neurological changes caused by neurodegenerative diseases.”

Once they develop the transgenic model for Huntington’s, Chan’s research team will use Yerkes’ new brain-imaging capabilities to monitor high-resolution magnetic resonance imaging scans while simultaneously conducting behavioral and cognitive studies. This novel approach will provide the researchers a broader, more comprehensive view of the disease than has ever been established.

“This study, along with other programs currently taking place at Yerkes, places us on the forefront of neuroscience research,” said Yerkes Director and study co-investigator Stuart Zola, who will lead the behavioral assessments. “Access to Emory’s department of human genetics, our collaborations with the department of neurology and our new brain-imaging center make Yerkes one of the few places in the world where this type of study can be done.”

“We have a better understanding of the genetic defects associated with Huntington’s disease than we do of other neurodegenerative diseases such as Parkinson’s,” said Steve Warren, W.P. Timmie Professor and chair of human genetics. “Establishing this nonhuman primate model for Huntington’s disease is critical to providing a foundation for studying the genetic causes of other neurodegenerative diseases.”

“This study allows Yerkes to lead the way toward a better understanding of neurodegeneration, and one day, toward developing better treatment options for an array of neurodegenerative diseases and disorders,” Zola said.

Before joining Yerkes, Chan was a staff scientist at the Oregon National Primate Research Center in Beaverton. While there, he and his colleagues created ANDi, the world’s first genetically modified nonhuman primate, to assist in developing primate models of human diseases, such as Alzheimer’s, diabetes and heart disease, that will offer more opportunities to test drugs, gene therapy and stem cell modification.

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