Application of stem cells to developmental modeling and cell-based therapy for neuromuscular and musculoskeletal disorders.
My current research focuses on developing stem cell technology for use in treating and modeling neuromuscular and musculoskeletal diseases. We have engaged in basic and translational studies using human neural progenitor cells, mesenchymal stem cells, and pluripotent stem cells to develop therapeutic strategies for neuromuscular diseases, primarily focusing on amyotrophic lateral sclerosis (ALS). ALS is a progressive, neurodegenerative disease in which motor neurons of the spinal cord and brain degenerate, causing paralysis and death due to respiratory failure.
On the translational front, my laboratory uses human stem cells for developing therapeutic strategies to treat ALS. Our current idea is to use stem cells to provide growth factor delivery using stem cells to the neuromuscular junction. This approach protects motor neurons by preventing the “dying back” of these cells from the muscle in a rat model of ALS.
Furthermore, our lab recently initiated a new project to establish skeletal muscle progenitor/stem cells derived from human pluripotent sources. Our culture method can produce skeletal muscle progenitor cells from human induced-pluripotent stem cells generated from both healthy donors and patients with neuromuscular disorders. This project will allow us to refine our ex vivo therapeutic approach for ALS and develop in vitro models of neuromuscular diseases.
In addition, our research team develops new imaging approaches for monitoring the location and survival of engrafted stem cells in the central nervous system. In preclinical and clinical trials of stem cell therapy, it is essential to investigate engrafted cell dynamics to understand patient effects.