Udai Pandey, PhD

Dr. Pandey’s laboratory is interested in understanding the molecular mechanisms of human neurological diseases such as amyotrophic lateral sclerosis (ALS) and traumatic brain injury. We have been using mammalian neuronal cells, Drosophila and patient-derived iPSC lines to examine the contribution of pathogenic mutations that lead to ALS pathogenesis. Several RNA-binding proteins have been implicated in human neurodegenerative diseases, such as polyglutamine expansion diseases and ALS. TDP-43, FUS, Matrin-3, VCP, and other RNA-binding proteins have been linked to ALS and suggest that defective RNA metabolism is central to ALS pathogenesis. The Pandey lab developed cellular and Drosophila models of ALS by expressing human and fly versions of disease-causing genes that regulate RNA metabolism. These models recapitulate several key features of the human disease, including neuromuscular junction defects, cytoplasmic accumulation of mutant protein, and behavioral defects. The Pandey lab is now performing genetic and small molecule screens to identify modifiers of neurodegenerative phenotypes in vivo. Discovery of these modifiers will enhance our understanding of mechanisms of ALS and will help in identifying new pathways for therapeutic drug development. The Pandey lab provides an exciting environment for training the next generation of physician-scientists and scientists interested in studying the molecular pathogenesis of human neurodegenerative diseases.