Allison D. Ebert, PhD

Therapeutic uses of stem cells and disease modeling for spinal muscular atrophy, amyotrophic lateral sclerosis, and congenital HCMV infection

My research interests are in the area of neurodegenerative diseases, both understanding the molecular basis for the disease progression and finding effective experimental therapies. My current research focuses on using induced pluripotent stem cells (iPSCs) derived from patient tissue to understand disease mechanisms and therapeutic intervention. Ongoing projects in my lab are investigating motor neuron malfunction and loss involved in spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS). We are using iPSCs derived from SMA and ALS patients to generate motor neurons, astrocytes, and microglia to determine mechanisms contributing to disease pathology. My lab has found that SMA astrocytes aberrantly upregulate a transcriptional network that increases neuroinflammation and that they have altered cell surface proteins that disrupt synapses. In ALS we have found that microglia and astrocytes together play a role in the accumulation of aggregated proteins and motor neuron loss. My lab then uses in vitro and in vivo gene therapy approaches to modulate astrocyte and microglial malfunction to improve motor neuron health and survival. Finally, we are investigating the cellular, molecular, and functional consequences of viral infection on brain development and neurodegeneration. Specifically, we use iPSC-derived neural progenitor cells, neurons, microglia, and cortical organoids to determine how infection with human cytomegalovirus alters neurodevelopment and test the impact of antiviral strategies.