Dr. Leonard Petrucelli, PhD - Mayo Clinic - Strategies For Complicated Neurodegenerative Disorders

Dr. Leonard Petrucelli, PhD ( https://www.mayo.edu/research/faculty/petrucelli-leonard-ph-d/bio-00027816 ), is a leading translational neuroscientist and Professor in the Department of Neuroscience at Mayo Clinic — Jacksonville, where he leads the Neurodegenerative Diseases Laboratory ( https://www.mayo.edu/research/labs/neurodegenerative-diseases/overview ).

Dr. Petrucelli’s lab seeks to understand and prevent neuronal degeneration and death in diseases characterized by abnormal protein aggregation and defects in RNA metabolism. His team uses a combination of cell biology, induced pluripotent stem cell technology, transcriptomics and mouse modeling to gain insight into disease mechanisms. They then leverage their findings to develop new therapeutic strategies and clinical tools tailored for these complicated disorders. Their current work focuses on a range of conditions, including Alzheimer's disease, frontotemporal dementia and amyotrophic lateral sclerosis (ALS).

Dr. Petrucelli trained in molecular and cellular biochemistry (Ph.D., Loyola University & Stritch School of Medicine) after earning a B.S. in Biology from Barry University, and he joined Mayo Clinic as a research fellow before rising to professor and department chair. His group combines human neuropathology, cell and molecular biology, and animal models to connect disease-linked genes and protein misprocessing to neuronal dysfunction and degeneration.

Scientifically, Dr. Petrucelli is best known for mechanistic work on aggregation-prone proteins and pathways that underlie neurodegeneration — including seminal contributions on TDP-43 biology, progranulin (GRN) dysfunction, and pathogenic mechanisms associated with C9orf72 repeat expansions — and for translating those findings toward biomarkers and therapeutic strategies. His lab’s work appears in high-impact journals and has helped define key pathogenic cascades now pursued by academic and industry drug-development programs.

Beyond his laboratory, Dr. Petrucelli serves in prominent advisory and leadership roles across the field: he is a voice on state and national Alzheimer’s advisory efforts, has advised disease-focused nonprofits and consortia (including Target ALS and others), and participates on scientific advisory boards for translational ventures. He is also an R35 (Research Program Award) recipient and an investigator with an extensive record of NIH-supported projects investigating mechanisms and potential interventions for FTD/ALS and related dementias.

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