Leukodystrophy Center

Pioneering Research

Researchers at the University of Minnesota continue to explore innovative ways to improve treatment success for patients with leukodystrophies. Our scientists lead the field in several areas of research and bringing these findings into clinical practice.

Current Projects

  • Researching basic biology of leukodystrophies and the implications for patients
  • Evaluating markers of disease activity, inflammation and oxidative stress in leukodystrophies
  • Exploring new treatment regimens for disorders such as GM1 gangliosidosis and Tay Sachs disease
  • Using intrathecal approaches (delivery of therapies to the fluid around the brain and spinal cord) to treat patients
  • Developing and testing approaches for gene therapy in leukodystrophies
  • Studying modification of transplant procedures to decrease disease progression
  • Testing means of expanding umbilical cord cells to enhance safety and effectiveness of transplant for leukodystrophies
  • Using transplantation to treat adults with leukodystrophies
  • Studying risk factors affecting outcomes following transplantation
  • Understanding late effects of bone marrow transplantation
  • Understanding risk of AMN (spinal cord disease) in post-transplant ALD patients