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About

Jeffrey Rothstein is a pioneering neuroscientist whose research has fundamentally advanced our understanding of neurodegenerative diseases, particularly amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). His major contributions center on the role of RNA metabolism and protein aggregation in motor neuron degeneration, and he is widely recognized for spearheading the discovery of TDP-43 pathology as a hallmark of ALS and FTD. Rothstein’s work has also been instrumental in developing cellular and animal models that have become essential tools for the field. Among his most cited works is the landmark 2017 paper "The Library of Integrated Network-Based Cellular Signatures NIH Program: System-Level Cataloging of Human Cells Response to Perturbations," which has garnered over 447 citations and reflects his leadership in large-scale, systems-level approaches to drug discovery. With a career spanning decades, Rothstein’s research has not only elucidated disease mechanisms but also paved the way for clinical trials targeting glutamate excitotoxicity and RNA dysregulation. His impact is evidenced by over 100,000 total citations, numerous awards including the Sheila Essey Award for ALS Research, and his role as a director of the Robert Packard Center for ALS Research at Johns Hopkins.