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UHN plays key role in ambitious project to identify new therapies for Rett Syndrome.
Posted On: April 20, 2015
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The first symptoms of Rett Syndrome occur between 6 and 18 months of age and include loss of motor skills such as the ability to purposefully use hands.
A new research partnership between UHN, the University of Toronto (UT) and the Hospital for Sick Children will search for new treatments for a rare disease known as Rett Syndrome. The disorder is an X chromosome-linked genetic disorder that primarily affects girls early in life and causes previously acquired skills, such as speech and motor skills, to be lost. Currently there is no cure for the disease.
With funding from the Ontario Brain Institute, the project will identify and test a group of top drug candidates to target a protein involved in the development of the disease.
TWRI Senior Scientist Dr. James Eubanks will serve as the UHN lead and will provide valuable experimental models of Rett Syndrome that were developed in his lab—models that will be instrumental in testing the compounds as potential treatments for the disease.
Dr. Donald Weaver, the Director of TWRI, comments, “This is a unique partnership that we are excited to be a part of. By leveraging the expertise, resources and innovations through the lab of our contributing researcher, Dr. James Eubanks, we hope to have progress against this disease in a way that will have an impact for patients and families that are affected by it.”
At UT, Dr. Aled Edwards’ group within the Structural Genomics Consortium (SGC) will provide the candidate molecules. The initial group of compounds that will serve as a starting point for researchers was identified through an innovative public-private agreement. The agreement enabled hundreds of academic institutions and ten leading pharmaceutical companies to share results globally.
With funding from the Ontario Brain Institute, the project will identify and test a group of top drug candidates to target a protein involved in the development of the disease.
TWRI Senior Scientist Dr. James Eubanks will serve as the UHN lead and will provide valuable experimental models of Rett Syndrome that were developed in his lab—models that will be instrumental in testing the compounds as potential treatments for the disease.
Dr. Donald Weaver, the Director of TWRI, comments, “This is a unique partnership that we are excited to be a part of. By leveraging the expertise, resources and innovations through the lab of our contributing researcher, Dr. James Eubanks, we hope to have progress against this disease in a way that will have an impact for patients and families that are affected by it.”
At UT, Dr. Aled Edwards’ group within the Structural Genomics Consortium (SGC) will provide the candidate molecules. The initial group of compounds that will serve as a starting point for researchers was identified through an innovative public-private agreement. The agreement enabled hundreds of academic institutions and ten leading pharmaceutical companies to share results globally.