We are grateful to our patients, their families, and the advocacy organizations that make our research possible. Together, we continue to make significant progress in the understanding and treatment of leukodystrophies.
Selected Research Projects
One of our goals at the University of Minnesota is to understand the mechanism of how and why the cerebral form of adrenoleukodystrophy (ALD) starts. As the largest transplant center in the world, we are in a unique position to answer this question. Our international experts want to use our biorepository and database to search for potential triggers of cerebral disease. They hope to investigate questions about biomarkers, environmental exposure, gene mutations, imaging, infection, and neuropsychology.
Another goal is to improve the “mouse model” of ALD. Currently, the mice that have the ALD gene mutation do not have cerebral inflammation. We are working on creating a better mouse model of the disease that mimics the cerebral disease of the boys more closely. This will enable us to test new and novel therapies faster and more effectively compared to the current testing performed on cells in a dish.
Knockout ALD organizes and hosts the Run for ALD, Cure the Boys 5K and 1 mile walk event in Maryland Heights, Missouri to raise funds for our ALD Research Fund. This year's event will take place on Saturday, November 16, 2019. To register, please complete this form. You can read Nicholas's story here.
The Calliope Joy Foundation supports our Leukodystrophy Center in many ways. TCJF has made it possible to produce two educational videos for ALD newborn screening. We have also received gifts for leukodystrophy research at our center. In addition, our patients have been given shirts, hats, and books!