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On to the next step

Because of philanthropic support, research focused on developing the first-ever treatment for spinocerebellar ataxia type 1 (SCA1) continues to move forward.

Working with Beverly Davidson, Ph.D., of the Children’s Hospital of Philadelphia, Harry Orr, Ph.D., and his University of Minnesota team are working to develop a gene therapy for SCA1. One method to treat dominantly inherited genetic disorders such as SCA1 is to remove the toxic gene product through a method known as RNA interference (RNAi). It’s thought that RNAi can cause the disease-encoding molecules to degrade and thereby stop expression of the toxic protein.

If proven effective, this therapy could potentially be used to treat other neurodegenerative diseases, such as Huntington’s disease and genetic forms of Alzheimer’s and Parkinson’s diseases, as well.

Up to this point, this research has been largely supported by philanthropy, particularly a $50,000 matching gift challenge from benefactors Richard and Maureen Schulze. Nearly 100 additional donors stepped forward to take part in the challenge, together raising another $66,932 for the project.

Research results from the first stage of this work were more promising than the researchers expected, which meant that they were able to move on to the next step faster than expected— using less money than expected. The team has carried over the remaining dollars to support the next step of their investigation, but they’ll need an additional $650,000 to fund it fully. “If the research continues to move forward at the rate it is now, we are optimistic that we’re potentially two to three years away from clinical trials for patients,” Orr says.

Philanthropy will continue to advance this project. For more information about the research or to make a gift, contact Tracy Ketchem of the University of Minnesota Foundation at 612-625-1906 or tketchem@umn.edu.

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