ISCRM faculty members Mark Bothwell, a Professor of Neurobiology & Biophysics and Yusha (Katie) Liu, an Assistant Professor of Surgery, have received prestigious awards from the John H. Tietze Foundation Trust that will help fuel promising research underway in their labs.
Creating a New Platform to Screen Drugs for Neuromuscular Diseases
Within the Neuromuscular Disease Research Group, The Bothwell Lab uses induced pluripotent stem cells to study neuromuscular diseases, like Charcot-Marie Tooth, ALS, and muscular dystrophies. While the team currently produces human motor neurons and skeletal muscle from stem cells, their models lack myelinating Schwann cells, which are essential for normal neuromuscular transmission, and which are the specific site of defects in the most common forms of Charcot-Marie Tooth disease. This shortcoming limits their ability to accurately identify neuromuscular disease mechanisms.
With funding from the 2026 John H. Tietze Stem Cell Scientist Award, the Bothwell Lab will test a hypothesis that the struggle to produce mature human myelinating Schwann cells is rooted in a failure to replicate the correct expression of genes known as EGR1, EGR2, and EGR3, which are master regulators of Schwann cell development. To address this limitation, the Bothwell Lab will use gene-editing techniques to produce stem cells in which expression of EGR2 and EGR3 can be artificially controlled by drug exposure. The team will then use RNA sequencing comparing cells resulting from that protocol to Schwann cells isolated from human nerves to optimize generation of myelinating Schwann cells.
Achieving formation of human neuromuscular junctions by myelinated motor nerve fibers in vitro will provide a powerful platform for preclinical screens of drugs for neuromuscular diseases.
Exploring the Use of Fat Stem Cells to Repair Damaged Nerves
For physicians attempting to treat patients with peripheral nerve injuries, one of the biggest challenges is the slow speed at which nerves regenerate. Scientists estimate that nerve fibers known as axons grow at a millimeter per day or an inch a month. That pace could mean multiple years for a nerve of the brachial plexus in the neck to grow to the wrist and hand. Meanwhile, as time passes, muscles impacted by a peripheral nerve injury gradually lose their ability to reestablish connection with the nerves. Keeping the window open longer could therefore help doctors maximize recovery for patients.
With funding from the Jaconette L. Tietze Young Scientist Award, Dr. Katie Liu will explore whether stem cells taken from a patient’s own fat tissue can help repair damaged nerves. These cells are known as adipose-derived mesenchymal stem cells (ADMSC)
This research will build on current efforts in the Liu Lab to develop therapeutics that could be applied at the time of surgery, which might allow for greater control over the regeneration process. Support will allow Liu and her team to use cells taken from liposuction tissues to examine why some patients’ stem cell products may be better at repairing nerves than others, and which proteins might be responsible.
The researchers believe the data generated through this work will point to protein biomarkers that correlate to the regenerative capacity of an individual’s fat cells and produce important insights about whether these cells can be used to treat peripheral nerve injuries. Furthermore, a better understanding of how the make-up of ADMSCs differ from person to person will provide the broader field of regenerative medicine with new options for studying and treating a wide range of diseases.