Grant to fund stem cell research aimed at helping patients with a type of paralysis
The Carter Foundation for Neurologic Research, Inc., awarded an $80,000 grant to Xue-Jun Li, PhD, for a research project to identify biomarkers and potential therapies for the most common type of early-onset spastic paraplegia. Dr. Li is the Michael A. Werckle Professor of Biomedical Sciences and co-director of the UICOMR Regenerative Medicine and Disability Laboratory.
Dr. Li and her team will study spastic paraplegia 3A (SPG3A), which is the result of a genetic mutation that causes the degeneration of a group of nerve cells in the brain that control muscle movement. This degeneration leads to stiffness and weakness of the leg and hip muscles and most cases are diagnosed in children before the age of 10.
In the laboratory, Dr. Li will use cells from SPG3A patients to generate pluripotent stem cells that can become patient-specific nerve cells with the genetic mutation, which her team can then study to learn more about the disease. They will also test therapeutic agents that could reverse the defects in such cells.
“Using RNA-sequencing analyses, this study will identify factors and pathways that are specifically altered in nerve cells derived from SPG3A patient stem cells with different mutations,” says Dr. Li. “Moreover, this study will examine and compare the efficacy of several classes of pre-clinical compounds to identify biomarkers and therapeutic agents for SPG3A. Together, this study will be valuable in identifying new targets and developing potential therapies for SPG3A.”